Improving Access to Gene Therapies: A Holistic View of Current Challenges and Future Policy Solutions in the United States

Gene therapies hold the promise of delivering groundbreaking improvements in health outcomes for previously intractable diseases. These therapies possess unique characteristics that differ from traditional small molecule or biologic treatments, posing new challenges for healthcare stakeholders. While previous analyses have predominantly focused on the payment challenges associated with emerging gene therapies, a more holistic examination reveals numerous obstacles that currently hinder optimal patient access. In the U.S., these include production and distribution issues, logistical and treatment administration challenges and coverage and reimbursement limitations imposed by U.S. healthcare payers. Several opportunities exist to address these challenges and improve patient access, including the use of appropriate value assessment methods, the development of innovative payment solutions, the removal of inappropriate access barriers imposed by payers and improving education and awareness regarding treatment availability, benefits and risks. 

What We Stand to Lose from Federal Research Funding Cuts

Washington is considering reducing annual funding for basic science research by roughly $15 billion, a 34% drop. There’s nothing wrong with reviewing all spending to ensure that taxpayer money is being well-spent during an era of crushing budget deficits. But draconian, across-the-board cuts in government-supported R&D would undermine our economy and national security — by weakening the tech transfer system that made America the global leader in innovation. As tech transfer professionals know — but some policymakers may not — both our public and private sectors play critical roles in funding research and driving the American innovation which is the envy of the world. The federal government is the largest sole funder of basic research, both at its own labs and at universities across the country. 

Pharma Companies Grow, Evolve Across New Jersey

In New Jersey, pharmaceutical companies operate from Princeton to Parsippany. These companies represent establishments spanning from 1849 to 2021, demonstrating the evolution of pharmaceutical operations in New Jersey. Here’s a closer look at some of them. Abbott Laboratories Pharmaceutical Products established operations in 1888, making it one of the companies with the longest presence. Johnson & Johnson employs 138,000 people worldwide. Merck. operates from Rahway with about 75,000 global employees. Bristol-Myers Squibb maintains its presence in Princeton with approximately 30,000 employees worldwide. Organon & Co. was acquired by Schering-Plough in 2007, then became part of Merck in 2009 and spun off as an independent company in 2021 — now operating from Jersey City with 10,000 employees. Sanofi relocated from Bridgewater to Morristown in May 2025, maintaining its 100,000-employee workforce.

Empowering National Security: The Women-Owned Company Redefining Antiviral Treatment

Pop Test Oncology LLC, operating as Palisades Therapeutics (PT), is proud to be at the forefront of medical innovation and national security. We were recently invited by leaders at the Department of Homeland Security’s Chemical, Biological, Radiological, Nuclear (CBRN) Division and the Medical CBRN Defense Consortium (MCDC) conference to present our cutting-edge Host Directed Antiviral platform — a recognition of our technology’s potential in safeguarding the nation’s health infrastructure against today's and tomorrow’s threats. The enthusiastic response from these pivotal government agencies reinforces the urgent need for novel solutions in medical countermeasures (MCMs). While major pharma remains cautious, PT, a nimble, women-owned enterprise, is seizing the opportunity to drive transformative advances.

BeOne Medicines: Advancing Innovation in Cancer Treatment Across New Jersey and America

Each year in the United States, nearly two million people are diagnosed with cancer. Globally, it’s the second leading cause of death — and by 2040, annual cancer cases are projected to surge to nearly 30 million, with more than 15 million deaths. BeOne Medicines is addressing this staggering challenge head-on. We are committed to uniting the worldwide community against cancer by delivering innovative medicines faster and expanding access for patients, wherever they live. A key pillar of our bold vision to eradicate cancer is firmly rooted in New Jersey. One year ago, we advanced that mission by opening our flagship U.S. biologics manufacturing and clinical R&D facility in Hopewell — a vital hub powering our efforts to build a next-generation company driven by innovation, access and speed.

BMS Manufacturing Facility Celebrates 120 Years of Innovation

BioNJ Member Bristol Myers Squibb recently hosted government officials on a tour of its labs and manufacturing spaces in New Brunswick where BMS employees have transformed bold science into life-changing medicines for over a century. The pharmaceutical company’s global hub for clinical supply manufacturing in oncology, immunology, cardiovascular and more, is celebrating 120 years of scientific innovation. From producing the world’s largest supply of penicillin in 1944 to today’s groundbreaking research that turns scientific discoveries into tangible medicines for clinical trials, the manufacturing facility continues to drive healthcare innovation. BioNJ, and others, had the opportunity to take an inside look at the cutting-edge work being done by the 2,400 BMS employees based in New Brunswick.

Presenting the 2025 People to Watch in Health Care

The NJBIZ “watch” lists are designed to identify individuals who contribute greatly to the success of their organizations ― and, in the case of health care, to the well-being of the State – but have yet to earn wide recognition for their efforts. Some, like Shlomi Madar and Vincent Mileto, are entrepreneurs with fascinating ideas. Dr. Madar runs SpotitEarly, a company dedicated to making early detection of cancer more widely available by using artificial intelligence to harness the well-known olfactory powers of dogs. And Dr. Mileto invented a new way to deliver medicines orally ― with a quick dissolving tablet that will be life-changing for patients who have difficulty swallowing normal tablets. Both were featured at the most recent BioNJ BioPartnering Conference, which is a great place to meet people worth watching.

NJBIZ Conversations: Orcosa Co-Founders Bryan Ridall and Vincent Mileto

Anyone who has ever experienced difficulty swallowing medication or who has struggled to help a child or pet take their prescriptions will welcome the work of Bryan Ridall, Vincent Mileto and their team at Orcosa. The Ewing-based company developed the The RITe Platform. The technology produces an easy-to-take orally disintegrating tablet that rapidly infuses an active ingredient through the tissue in the cheek — enabling therapeutic effects quickly and efficiently. The medication dissolves in seconds, without swallowing, avoiding the problems that can plague human and animal patients with certain conditions. The company has received positive test results from its Oravexx pain treatment. The non-addictive proprietary cannabidiol compound is currently being studied in clinical trials. The company recently participated in BioNJ’s BioPartnering Conference in Jersey City.

PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living With Phenylketonuria (PKU)

Warren-based BioNJ Member PTC Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU. The FDA approval is based on the evidence of significant efficacy and safety from the Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study. SEPHIENCE was recently granted marketing authorization by the European Commission. Review of approval applications is ongoing in several other countries including Japan and Brazil.

FDA Approves SKYTROFA® (Lonapegsomatropin-tcgd) for the Once-Weekly Treatment of Adults With Growth Hormone Deficiency

BioNJ Member Ascendis Pharma A/S, with a site in Princeton, announced that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA® (lonapegsomatropin-tcgd; developed as TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD), a rare disorder resulting from decreased or total loss of growth hormone production. Lonapegsomatropin (approved by the FDA in 2021 for the treatment of pediatric GHD) is a prodrug of somatropin (human growth hormone, or hGH) administered once weekly, providing sustained release of active, unmodified somatropin. The FDA’s approval of SKYTROFA for adult GHD was based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) clinical trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily somatropin in adults with GHD. “Our market research shows SKYTROFA is the treatment of choice for pediatric GHD among patients and physicians, and we are pleased to expand its availability in the U.S. for the treatment of adults initiating therapy or switching from another growth hormone therapy,” said Jan Mikkelsen, President and CEO at Ascendis Pharma.

BeOne Medicines Receives Positive CHMP Opinion for TEVIMBRA® in Neoadjuvant/Adjuvant NSCLC Treatment

Hopewell-based BioNJ Member BeOne Medicines Ltd. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion recommending approval of TEVIMBRA® (tislelizumab), in combination with platinum-containing chemotherapy as neoadjuvant treatment and then continued as monotherapy as adjuvant treatment, for the treatment of adult patients with resectable non-small cell lung cancer (NSCLC) at high risk of recurrence. This recommendation is based on the Phase 3 RATIONALE-315 study. RATIONALE-315 is a double-blind, placebo-controlled, multicenter, Phase 3 study that randomized 453 patients with resectable NSCLC 1:1 to receive either TEVIMBRA plus platinum-based doublet chemotherapy as neoadjuvant treatment followed by TEVIMBRA as adjuvant treatment or placebo plus platinum-based doublet chemotherapy as neoadjuvant treatment followed by placebo as adjuvant treatment. 

BeOne Medicines Receives PRIME Designation from the European Medicines Agency for BGB-16673 in Waldenstrom’s Macroglobulinemia

Hopewell-based BioNJ Member BeOne Medicines Ltd. announced that the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) designation to the Company’s investigational Bruton’s tyrosine kinase (BTK) degrader, BGB-16673, for the treatment of patients with Waldenstrom’s macroglobulinemia (WM) previously treated with a BTK inhibitor. In addition to the PRIME designation, the EMA’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on the EU Orphan Drug Designation (ODD) application for BGB-16673 in WM. A final decision is anticipated in the coming weeks. The U.S. Food and Drug Administration (FDA) also granted Fast Track Designation to BGB-16673 for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and adult patients with R/R mantle cell lymphoma (MCL). The EMA’s CHMP granted PRIME designation to BGB-16673 based on data demonstrating its novel mechanism and anti-tumor activity in B-cell malignancies. 

European Commission Approves TEVIMBRA in Combination With Chemotherapy as a First-Line Treatment for Nasopharyngeal Carcinoma

Hopewell-based BioNJ Member BeOne Medicines Ltd. announced that the European Commission has approved TEVIMBRA (tislelizumab), in combination with gemcitabine and cisplatin, for the first-line treatment of adult patients with metastatic or recurrent nasopharyngeal carcinoma (NPC), not amenable to curative surgery or radiotherapy. Nasopharyngeal carcinoma is a rare cancer in which malignant cells form in the nasopharynx, the upper part of the throat located behind the nose. The approval in NPC is based on results of RATIONALE-309 (NCT03924986), a double-blind, placebo-controlled, multicenter, Phase 3 study, which randomized 263 treatment-naïve patients who received either TEVIMBRA in combination with gemcitabine plus cisplatin or placebo in combination with gemcitabine plus cisplatin.

Biological Efficacy Demonstrated in a Phase 2 Clinical Trial of SGX945 for the Treatment of Behçet's Disease

Princeton-based BioNJ Member Soligenix, Inc. announced that it has completed its Phase 2a proof of concept study evaluating SGX945 (dusquetide) in the treatment of Behçet's Disease and achieved the study objective of demonstrating biological efficacy. The Phase 2a study was an open-label study designed to be highly comparable (e.g., study endpoints, inclusion-exclusion criteria) to the published Phase 3 study of apremilast (Otezla®) used to support marketing approval for oral ulcers in Behçet's disease. SGX945 outcomes were compared to both the apremilast and placebo arms in this Phase 3 study. Over 4 weeks of treatment, the area under the curve (AUC; a composite measurement of both peak number of oral ulcers and the time to resolution of the oral ulcers), average number of oral ulcers, and improvements in oral pain for SGX945 were similar to outcomes obtained in the apremilast study.

Taiho Oncology and Taiho Pharmaceutical Announce U.S. FDA Acceptance of Supplemental New Drug Application for INQOVI in Combination With Venetoclax to Treat Patients With Acute Myeloid Leukemia

Princeton-based BioNJ Member Taiho Oncology, Inc., and Taiho Pharmaceutical Co., Ltd., announced that the U.S. Food and Drug Administration (FDA) has accepted their supplemental new drug application (sNDA) for INQOVI (decitabine and cedazuridine) plus venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. The FDA assigned a standard review with a Prescription Drug User Fee Act (PDUFA) target action date of February 25, 2026. The sNDA is supported by results from ASCERTAIN-V, a Phase 2b study of INQOVI plus venetoclax in 101 adult patients with newly diagnosed AML who were ineligible for intensive induction chemotherapy. INQOVI is an orally administrated hypomethylating regimen, currently indicated in the U.S. for the treatment of adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).

Boehringer Ingelheim and LEO Pharma Enter Partnership to Commercialize and Further Develop SPEVIGO® (spesolimab)

BioNJ Member Boehringer Ingelheim, with a site in North Brunswick, and Madison-based LEO Pharma announced an exclusive global license and transfer agreement to commercialize and advance the development of SPEVIGO® (spesolimab). SPEVIGO® is an innovative, humanized, and selective monoclonal antibody that targets and blocks the activation of the interleukin-36 (IL-36) receptor - a key signaling pathway in the immune system implicated in the pathogenesis of several autoinflammatory diseases, including generalized pustular psoriasis (GPP). This partnership extends beyond GPP, with an opportunity to investigate the potential of spesolimab in additional skin conditions with high unmet medical need in which IL-36 is implicated. Under the terms of the agreement, LEO Pharma will be responsible for commercialization and further development of SPEVIGO® leveraging its global commercial platform within medical dermatology to raise disease awareness and secure access for patients with GPP. 

Tevogen Signs LOI for New In-House Cell Therapy Manufacturing Facility

Warren-based BioNJ Member Tevogen signed a letter of intent to lease a 17,428-square-foot facility that should support the company’s cell therapy manufacturing operations. The space, formerly occupied by Pfizer and Cordis, a previous division of Johnson & Johnson, aligns with Tevogen’s goals of accelerating clinical development while maintaining cost efficiency and scalability. “Our continued investment in infrastructure reflects our commitment to delivering innovative and accessible T cell therapies,” Dr. Ryan Saadi, CEO of Tevogen Bio said. “This facility has potential to efficiently scale our manufacturing as we advance our pipeline toward commercialization and will serve as a bridge as we enter the early stages of developing our own custom-built manufacturing facility.”

Made Scientific, Sentinel BioTherapeutics Announce Manufacturing Partnership to Advance Cell Therapy Program

Princeton-based BioNJ Member Made Scientific and Sentinel BioTherapeutics announced a strategic manufacturing partnership to support the Phase I/II clinical development of Sentinel’s SENT-001. SENT-001 is an allogeneic, encapsulated cell therapy leveraging genetically engineered allogeneic ARPE-19 cells to enable delivery of native human IL-2 (interleukin-2) to the peritoneal or pleural cavities. The therapy was previously evaluated under a multicenter, open-label Phase I study for patients with high-grade serous adenocarcinoma of the ovary, fallopian tube, or primary peritoneum. Sentinel BioTherapeutics is the first spinout of RBL LLC, a Houston-based biotech accelerator focused on accelerating novel therapeutic delivery technologies to clinics. As part of the collaboration, Made Scientific will establish a master cell bank and support both process optimization and clinical drug product manufacturing of SENT-001 as an off-the-shelf product. 

Bristol Myers Squibb and Bain Capital Create New Company Dedicated to Developing Innovative Immunology Therapies that Address the Unmet Medical Needs of Patients

Princeton-based BioNJ Member Bristol Myers Squibb and Bain Capital announced the creation of a new independent biopharmaceutical company focused on developing new therapies for autoimmune diseases that address significant unmet needs of patients. The newly formed company launches with five immunology assets in-licensed from BMS and a $300 million financing commitment that was led by Bain Capital. The NewCo has a broad pipeline consisting of three clinical-stage and two Phase 1-ready investigational medicines that each target promising mechanisms in autoimmune diseases. The most advanced assets in the NewCo’s portfolio are afimetoran, an oral, potential best-in-class TLR7/8 inhibitor that is currently being studied in a Phase 2 clinical trial for systemic lupus erythematosus (SLE), and BMS-986322, an oral TYK2 inhibitor, which successfully established proof-of-concept in a positive plaque psoriasis Phase 2 trial.

Bristol Myers Squibb’s Supplemental New Drug Application (sNDA) for Sotyktu (deucravacitinib) for the Treatment of Adults With Active Psoriatic Arthritis Accepted for Review Across Four Regions Globally

Princeton-based BioNJ Member Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for Sotyktu (deucravacitinib) for the treatment of adults with active psoriatic arthritis. This latest regulatory milestone is in addition to the sNDA acceptances by China’s Center for Drug Evaluation of National Medical Products Administration and Japan's Ministry of Health, Labour and Welfare for Sotyktu for the treatment of adults with active psoriatic arthritis. The European Medicines Agency has also validated Bristol Myers Squibb's Type II variation application to expand the indication for Sotyktu to include this disease. Sotyktu received approval from the FDA in 2022 for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. 

Bristol Myers Squibb and Pfizer Announce Direct-to-Patient Eliquis® (apixaban) Option

The Princeton-based BioNJ Member Bristol Myers Squibb and BioNJ Member Pfizer, with a site in Peapack, Alliance announced a new direct-to-patient option for purchasing Eliquis® (apixaban) via our Alliance’s patient resource Eliquis 360 Support. The offering provides an opportunity for uninsured, underinsured, or self-pay patients to significantly lower their out-of-pocket costs for this critical medicine. Eliquis is relied upon by millions of Americans daily and this program will provide a simple and transparent way to access it. Beginning September 8, eligible U.S. patients with an Eliquis prescription may purchase the medicine directly through Eliquis 360 Support and will pay a discounted rate of more than 40% less than the current list price. 

Pfizer and BioNTech Receive Positive CHMP Opinion for LP.8.1-adapted COVID-19 Vaccine in the European Union

BioNJ Member Pfizer Inc., with a site in Peapack, and BioNTech SE announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for the companies’ LP.8.1-adapted monovalent COVID-19 vaccine (COMIRNATY® LP.8.1) for active immunization to prevent COVID-19 caused by SARS-CoV-2 in individuals 6 months of age and older. The adaptation is based on the recommendation from the EMA’s Emergency Task Force (ETF) to update COVID-19 vaccines to target the LP.8.1 variant for the 2025-2026 season. The ETF stated that “targeting LP.8.1 will help maintain the effectiveness of the vaccines as SARS-CoV-2 continues to evolve.” The CHMP’s recommendation will be reviewed by the European Commission (EC), which is expected to make its final decision soon.

XTANDI Plus Leuprolide Significantly Improves Survival Outcomes in Men With Non-Metastatic Hormone-Sensitive Prostate Cancer With High-Risk Biochemical Recurrence

BioNJ Member Pfizer Inc., with a site in Peapack, and Astellas Pharma Inc. announced positive topline results from the overall survival (OS) analysis from the Phase 3 EMBARK study evaluating XTANDI® (enzalutamide), in combination with leuprolide and as a monotherapy, in men with non-metastatic hormone-sensitive prostate cancer (nmHSPC; also known as nonmetastatic castration-sensitive prostate cancer or nmCSPC) with biochemical recurrence (BCR) at high risk for metastasis. For patients treated with XTANDI plus leuprolide versus placebo plus leuprolide, EMBARK met the key secondary endpoint with a statistically significant and clinically meaningful improvement in OS. Results also showed a favorable trend towards improved OS for patients treated with XTANDI monotherapy versus placebo plus leuprolide, however the difference did not reach statistical significance.

Sanofi’s SAR446523, a GPRC5D Monoclonal Antibody, Earns Orphan Drug Designation in the U.S. for Multiple Myeloma

Morristown-based BioNJ Member Sanofi announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SAR446523, an IgG1-based Antibody-Dependent Cellular Cytotoxicity-enhanced (ADCC) monoclonal antibody (mAb) targeting G-protein coupled receptor family C group 5 member D (GPRC5D) for the potential treatment of patients with relapsed or refractory multiple myeloma (R/R MM). GPRC5D is highly expressed on plasma cells in MM patients, with low expression in healthy tissues. The FDA grants orphan drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. “The orphan drug designation is a significant milestone in our ongoing efforts to develop innovative treatments in multiple myeloma,” said Alyssa Johnsen, M.D., Ph.D., Global Therapeutic Area Head, Immunology and Oncology Development at Sanofi. 

Sanofi’s Sarclisa Approved in the EU for the Treatment of Transplant-Eligible Newly Diagnosed Multiple Myeloma

Following the positive opinion by the European Medicines Agency's Committee for Medicinal Products for Human Use on June 19, 2025, the European Commission has approved Morristown-based BioNJ Member Sanofi’s Sarclisa in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for the induction treatment of adult patients with newly diagnosed multiple myeloma (NDMM) who are eligible for autologous stem cell transplant. The approval is based on results from part one of the two-part, double-randomized, German-speaking Myeloma Multicenter Group (GMMG)-HD7 Phase 3 study (clinical study identifier: NCT03617731), which was designed to independently assess the effects of Sarclisa during the induction and maintenance phases. Sarclisa-VRd demonstrated a deep and rapid response in transplant-eligible (TE) NDMM patients compared to VRd alone, reflected by a statistically significant minimal residual disease (MRD) negativity benefit at the end of the 18-week induction period, which was the primary endpoint of part one.

Sanofi to Acquire Vicebio, Expanding Respiratory Vaccines Pipeline

Morristown-based BioNJ Member Sanofi announces it has entered into an agreement to acquire Vicebio Ltd, a privately held biotechnology company headquartered in London, UK. The acquisition brings an early stage combination vaccine candidate for respiratory syncytial virus (RSV) and human metapneumovirus (hMPV), both respiratory viruses, and expands the capabilities in vaccine design and development with Vicebio’s ‘Molecular Clamp’ technology. The vaccine candidate complements Sanofi’s position in the respiratory vaccines space where the company is present in flu and RSV prevention. It allows Sanofi to offer increased physician and patient choice in RSV and hMPV by adding a non-mRNA vaccine to its pipeline. In addition, the acquisition adds ‘Molecular Clamp’, an innovative technology that stabilizes viral proteins in their native shape, enabling the immune system to recognize and respond to them more effectively. 

GSK and Hengrui Pharma Enter Agreements to Develop Up to 12 Innovative Medicines Across Respiratory, Immunology & Inflammation and Oncology

Warren-based BioNJ Member GSK plc announced it has entered into agreements with Hengrui Pharma (600276.SH; 01276.HK) to develop up to 12 innovative medicines, adding significant new growth opportunities to the company beyond 2031. The programmes were selected to complement GSK’s extensive Respiratory, Immunology & Inflammation (RI&I) and Oncology pipeline, and assessed for their potential best- or first-in class profiles. GSK will pay $500 million in upfront fees across the agreements. The agreements include an exclusive worldwide license (excluding mainland China, Hong Kong, Macau and Taiwan) for a potential best-in-class, PDE3/4 inhibitor (HRS-9821) in clinical development for the treatment of chronic obstructive pulmonary disease (COPD) as an add-on maintenance treatment, irrespective of background therapy. 

Blenrep (belantamab mafodotin) Combinations Approved in EU for Treatment of Relapsed/Refractory Multiple Myeloma

Warren-based BioNJ Member GSK plc announced the approval of Blenrep in the European Union (EU) for the treatment of adults with relapsed or refractory multiple myeloma in combination with bortezomib plus dexamethasone (BVd) in patients who have received at least one prior therapy, and in combination with pomalidomide plus dexamethasone (BPd) in patients who have received at least one prior therapy including lenalidomide. The approval is based on superior efficacy results demonstrated by Blenrep combinations in the pivotal DREAMM-7 and DREAMM-8 phase III trials in relapsed or refractory multiple myeloma. These include statistically significant and clinically meaningful progression-free survival (PFS) for Blenrep combinations versus triplet standard of care combinations in both trials and overall survival (OS) versus a daratumumab-based triplet in DREAMM-7. 

U.S. FDA Approves GSK’s Shingrix in a Prefilled Syringe Presentation

Warren-based BioNJ Member GSK plc announced that the U.S. Food and Drug Administration (FDA) has approved a prefilled syringe presentation of Shingrix (GSK’s Recombinant Zoster Vaccine or RZV) for the prevention of shingles (herpes zoster). The new prefilled syringe removes the need to reconstitute separate vials prior to administration, simplifying the vaccine administration process for healthcare professionals. The existing vaccine presentation consists of two vials, a lyophilised (powder) antigen and a liquid adjuvant, which healthcare professionals combine prior to administering. The approval of the new presentation is based on data demonstrating technical comparability between the new and existing vaccine presentation.

Lilly's Mounjaro (tirzepatide), a GIP/GLP-1 Dual Agonist, Demonstrated Cardiovascular Protection in Landmark Head-to-Head Trial, Reinforcing its Benefit in Patients With Type 2 Diabetes and Heart Disease

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced topline results from SURPASS-CVOT, a first-of-its-kind head-to-head Phase 3 cardiovascular outcomes trial comparing two incretin therapies in adults with type 2 diabetes and established atherosclerotic cardiovascular disease. Mounjaro (tirzepatide), a GIP/GLP-1 dual receptor agonist, was compared to Trulicity (dulaglutide), a GLP-1 receptor agonist that showed a definitive cardiovascular benefit in the REWIND study. In SURPASS-CVOT, Mounjaro achieved the primary objective by demonstrating a non-inferior rate of major adverse cardiovascular events (MACE-3), including cardiovascular death, heart attack or stroke vs. Trulicity. In addition, while not controlled for multiplicity-adjusted type-1 error, Mounjaro showed improvements on key measures of A1C, weight, renal function and all-cause mortality. T

Lilly's Kisunla (donanemab-azbt) Showed Growing Benefit Over Three Years in Early Symptomatic Alzheimer's Disease

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced results from the long-term extension (LTE) of the Phase 3 TRAILBLAZER-ALZ 2 study showing that participants treated with Kisunla (donanemab-azbt) demonstrated slowing of decline, a benefit that continued to grow over three years compared to an untreated external cohort from the Alzheimer's Disease Neuroimaging Initiative (ADNI). Participants in the study who started treatment later still saw benefit. However, earlier initiation of Kisunla in study participants significantly reduced the risk of progression to the next stage of the disease compared to those who received Kisunla treatment later. The TRAILBLAZER-ALZ 2 LTE study was a Phase 3, double-blind extension of the original TRAILBLAZER-ALZ 2 trial, evaluating the efficacy and safety of Kisunla in individuals with early symptomatic Alzheimer's disease. Participants originally treated with Kisunla either continued treatment or were switched to placebo, while those initially on placebo began Kisunla in a blinded manner. 

Donanemab Receives Positive Opinion from the Committee for Medicinal Products for Human Use (CHMP) in Early Symptomatic Alzheimer's Disease

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending donanemab for the treatment of early symptomatic Alzheimer's disease in adults with confirmed amyloid pathology who are apolipoprotein E ε4 (ApoE4) heterozygotes or non-carriers. The European Commission is expected to make a regulatory decision on donanemab in the coming months. Alzheimer's disease currently affects as many as 6.9 million people in Europe, with this figure expected to almost double by 2050 as aging populations continue to increase. Approximately one-third of individuals with mild cognitive impairment or mild dementia due to Alzheimer's disease progress to the next clinical stage of disease in one year.

Lilly's Jaypirca (pirtobrutinib), the First and Only Approved Non-Covalent (reversible) BTK Inhibitor, Met its Primary Endpoint in a Head-to-Head Phase 3 Trial Versus Imbruvica (ibrutinib) in CLL/SLL

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced positive topline results from the Phase 3 BRUIN CLL-314 clinical trial of Jaypirca (pirtobrutinib), a non-covalent (reversible) Bruton's tyrosine kinase (BTK) inhibitor, versus Imbruvica (ibrutinib), a covalent BTK inhibitor, in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL). This study enrolled patients with treatment-naïve CLL/SLL and those who had been previously treated but were BTK inhibitor-naïve. The study met its primary endpoint of non-inferiority on overall response rate (ORR) as assessed by an independent review committee (IRC) in both the pre-treated and intent-to-treat populations. ORR favored pirtobrutinib with a nominal P-value for superiority1 (p <0.05). Progression free survival (PFS), a key secondary endpoint, was not yet mature at this analysis, but was trending in favor of pirtobrutinib.

Johnson & Johnson Files With U.S. FDA to Include New Evidence in TREMFYA® (guselkumab) Label as the Only IL-23 Inhibitor to Demonstrate Significant Inhibition of Joint Structural Damage in Active Psoriatic Arthritis

New Brunswick-based BioNJ Member Johnson & Johnson announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking approval to include new evidence in the TREMFYA^® (guselkumab) label for the inhibition of progression of structural damage in adults with active psoriatic arthritis (PsA). The submission is supported by the Phase 3b APEX study in patients with active PsA, which achieved both its primary endpoint of reducing joint symptoms (ACR20) and its major secondary endpoint of inhibited progression of structural damage as measured by change in the modified van der Heijde-Sharp (vdH-S) score at 24 weeks, compared to placebo in bio-naïve patients. Data from the APEX study were consistent with the well-established safety profile of TREMFYA^®.

Johnson & Johnson Seeks First Icotrokinra U.S. FDA Approval Aiming to Revolutionize Treatment Paradigm for Adults and Adolescents With Plaque Psoriasis

New Brunswick-based BioNJ Member Johnson & Johnson announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking the first approval of icotrokinra, a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor for the treatment of adults and pediatric patients 12 years of age and older with moderate to severe plaque psoriasis (PsO). Icotrokinra is uniquely designed to block the IL-23 receptor, which underpins the inflammatory response in plaque PsO and offers potential in other IL-23-mediated diseases. The application included data from four pivotal Phase 3 studies conducted as part of the ICONIC clinical development program, including ICONIC-LEAD, ICONIC-TOTAL and ICONIC-ADVANCE 1 & ICONIC-ADVANCE 2. 

Johnson & Johnson Announces Strategic Agreement With Pacira BioSciences to Expand Early Intervention Offering for Osteoarthritis of the Knee

New Brunswick-based BioNJ Member Johnson & Johnson MedTech announced a strategic co-promotion agreement with Pacira BioSciences, Inc., the industry leader in the delivery of innovative, non-opioid pain therapies. The agreement expands the company’s Early Intervention portfolio with ZILRETTA® (triamcinolone acetonide extended-release injectable suspension) an extended-release injectable for treatment of pain related to osteoarthritis (OA) of the knee. It also includes shared professional education and engagement initiatives to support awareness of non-surgical options earlier in the treatment process. Osteoarthritis is a progressive, degenerative disease that affects more than 30 million adults in the United States alone, with knee OA being one of the most common and debilitating forms. Despite a wide range of available treatments, many patients continue to experience pain and mobility limitations, highlighting the need for more effective, longer-lasting solutions earlier in the care pathway.

European Regulatory Authority Adopts Positive Opinion for Novo Nordisk’s Alhemo® (concizumab), Recommending Label Expansion to Treat Haemophilia A and B Without Inhibitors

Plainsboro-based BioNJ Member Novo Nordisk announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending an update of the Alhemo® (concizumab) label to include the treatment of severe haemophilia A and moderate or severe haemophilia B without inhibitors. The positive CHMP opinion is based on the results from the Phase 3 explorer8 trial, which met its primary endpoint. The results demonstrated that Alhemo® prophylaxis compared with no prophylaxis treatment led to an 86% reduction and 79% reduction in treated spontaneous and traumatic bleeds for patients living with haemophilia A without inhibitors and haemophilia B without inhibitors, respectively. In this trial, Alhemo® showed a favourable safety profile in patients with haemophilia A and B without inhibitors. Patient-Reported Outcome (PRO) data from the explorer8 study further indicated an improvement in health-related quality of life and reduction in treatment burden with Alhemo® treatment compared with no prophylaxis.

New Data Presented at AAIC Demonstrates Investigational LEQEMBI® (lecanemab-irmb) 360 mg Subcutaneous Maintenance Dosing Could Offer a New Option for Ongoing Treatment of Early Alzheimer’s Disease

Nutley-based BioNJ Member Eisai Co., Ltd. and Biogen Inc. announced results on investigational maintenance therapy with subcutaneous autoinjector (SC-AI) of lecanemab-irmb (U.S. brand name: LEQEMBI®), an anti-amyloid beta (Aβ) protofibril* antibody for the treatment of early Alzheimer’s disease (AD). Only lecanemab fights AD in two ways— targeting both protofibrils and plaque, which can impact tau accumulation downstream. Due to the reaccumulation of AD biomarkers and return to placebo rate of decline after therapy is stopped, Eisai is investigating a new lecanemab SC maintenance treatment option following 18 months of IV therapy so patients can continue to fight this progressive, relentless disease. Clinical trials of lecanemab SC were conducted as a sub-study of the open-label extension (OLE) following the core Phase 3 Clarity AD study in individuals with early AD, to evaluate a range of doses administered by SC vial or autoinjector.

URECE (Dotinurad) Launched in China as a Treatment for Gout

Nutley-based BioNJ Member Eisai Co., Ltd. announced that it has launched URECE in China as a treatment for Gout. URECE was approved by the National Medical Products Administration in China as a treatment for gout patients with hyperuricemia in December 2024. URECE is a new therapeutic medicine for gout and hyperuricemia discovered by FUJI YAKUHIN. It suppresses uric acid reabsorption and lowers blood uric acid levels by selectively inhibiting the urate transporter (URAT1) related to reabsorption of uric acid in the kidney. Eisai concluded a license agreement with FUJI YAKUHIN for China and five ASEAN member states: Indonesia, Malaysia, Myanmar, the Philippines and Thailand, granting exclusive development and distribution rights in these countries.

Gilead Receives Positive CHMP Opinions Under Accelerated Review From European Medicines Agency for Twice-Yearly Lenacapavir for HIV Prevention

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion under accelerated review recommending lenacapavir—the company’s injectable HIV-1 capsid inhibitor—for use as pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 in adults and adolescents with increased HIV-1 acquisition risk. The final European Commission (EC) decision is expected later this year, and, if approved, lenacapavir will be marketed in the European Union (EU) under the trade name Yeytuo®. The marketing authorization application (MAA) recommendation will now be reviewed by the EC as it evaluates lenacapavir as a potential new preventative strategy against HIV in all 27 EU Member States, as well as Norway, Iceland and Liechtenstein. 

In Collaboration With the Gates Foundation, Merck Advances MK-8527 Pre-Exposure Prophylaxis (PrEP) Clinical Trials Globally

Rahway-based BioNJ Member Merck & Co. announced the initiation of the EXPrESSIVE Phase 3 clinical trials, evaluating the safety and efficacy of MK-8527, an investigational once-monthly, oral nucleoside reverse transcriptase translocation inhibitor (NRTTI) for HIV pre-exposure prophylaxis (PrEP). The EXPrESSIVE-11 (MK-8527-011, NCT 07044297) trial will evaluate the safety and efficacy of MK-8527 among people with greater likelihood of HIV-1 exposure in 16 countries and will begin enrolling in August 2025. In collaboration with the Gates Foundation, the EXPrESSIVE-10 (MK-8527-010) trial will evaluate the safety and efficacy of MK-8527 in women and adolescent girls in sub-Saharan Africa and will begin enrolling in the next few months. The decision to initiate the Phase 3 clinical trial program was supported by the results of a double-blind, multicenter, Phase 2 trial (MK-8527-007, NCT 06045507) examining the safety and pharmacokinetics of MK-8527.

LEO Pharma Announces FDA Approval of ANZUPGO® (delgocitinib) Cream in the U.S.

Madison-based LEO Pharma announced that the U.S. Food and Drug Administration (FDA) has approved ANZUPGO® (delgocitinib) cream (20 mg/g) for the topical treatment of moderate-to-severe chronic hand eczema (CHE) in adults who have had an inadequate response to, or for whom topical corticosteroids are not advisable. ANZUPGO is an innovative steroid-free, topical pan-Janus kinase (JAK) inhibitor for adults with CHE.¹ ANZUPGO inhibits the JAK-STAT pathway, specifically blocking the activity of JAK1, JAK2, JAK3, and tyrosine kinase 2 (TYK2), and suppresses the various inflammatory responses that play a key role in the onset and subsequent flares of CHE. The FDA approval of ANZUPGO marks a significant milestone in LEO Pharma’s strategy to expand its presence in the U.S. market and deliver purposeful innovation in skin health.

Aptar Pharma Bolsters Clinical Trial Capabilities With Acquisition of Strategic Materials Manufacturing in Boonton

Aptar Pharma has acquired the clinical trial materials manufacturing capabilities of Mod3 Pharma (formerly Enteris Biopharma) from SWK Holdings. Aptar will now operate an FDA-inspected, state-of-the-art facility in Boonton. The site includes cGMP cleanrooms, high-potency Active Pharmaceutical Ingredient (API) suites, biologics capabilities, and advanced fill-finish technologies-fully aligned with Aptar Pharma’s drug delivery solutions portfolio. The move addresses market demand for clinical trial material supply services, particularly for Phase 1 and 2 cGMP fill and finish services for orally inhaled nasal drug products (OINDPs). “By integrating Phase 1 and 2 clinical manufacturing capabilities, we’re not just expanding our technical footprint ― we’re deepening our commitment to customers seeking a seamless and accelerated path to market,” Gael Touya, President of Aptar Pharma, said.

Sun Pharma’s Phase 3 Clinical Studies Evaluating Tildrakizumab 100 mg (ILUMYA®) in Active Psoriatic Arthritis Meet Their Primary Endpoint

Princeton-based Sun Pharmaceutical Industries Limited announced top-line results from two Phase 3 clinical studies evaluating the efficacy and safety of tildrakizumab 100 mg (ILUMYA®) administered over 24 weeks for treatment of active psoriatic arthritis. Treatment with tildrakizumab 100 mg (ILUMYA) resulted in greater improvements in PsA signs and symptoms at Week 24 compared to treatment with placebo. Both the INSPIRE-1 and INSPIRE-2 studies achieved the primary endpoint, with a higher proportion of patients in the INSPIRE-1 and INSPIRE-2 studies treated with tildrakizumab achieving ACR20 responses at week 24, compared to those receiving placebo (p < 0.05). Safety data in the studies was consistent with the well-documented safety profile of ILUMYA, which is approved for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy.

UroGen Announces Five-Year Long-Term Extension Study of the OPTIMA II Trial Demonstrates Long-Term Durability of Response to ZUSDURI™ in Patients With Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

Princeton-based UroGen Pharma Ltd. announced the publication of results of a five-year long-term extension study of the Phase 2b OPTIMA II trial evaluating ZUSDURI (mitomycin) for intravesical solution in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) demonstrating durable, long-term complete responses (CRs) in patients who initially achieved a CR following treatment with ZUSDURI. Among the 41 patients who achieved CR at three months post-treatment with ZUSDURI in the OPTIMA II trial, 25 remained in CR at 12 months and 17 entered the long-term follow-up study. For the 41 patients achieving CR at three months, the median Kaplan-Meier estimate of duration of response (DOR) was 24.2 months (95% CI 9.7, 42.1) with a median follow-up of 35.8 months.

UroGen Announces Publication of Phase 3b Study Results Demonstrating the Feasibility of Home Instillation of ZUSDURI™ for Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer in Reviews in Urology

Princeton-based UroGen Pharma Ltd. announced the publication in Reviews in Urology of results from a Phase 3b study evaluating the feasibility of administering ZUSDURI™ (mitomycin) for intravesical solution (formerly known as UGN-102) in the home setting. The study, titled "Home Instillation of UGN-102 for Primary Chemoablation of Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer: A Single-Arm, Open-Label, Phase 3b Trial," demonstrated that trained home health professionals (HHPs) can safely and effectively administer ZUSDURI outside of a traditional clinical setting. The study assessed the feasibility, safety, and early efficacy of at-home instillations of ZUSDURI in patients with recurrent LG-IR-NMIBC. Six of eight patients (75%) completed all six scheduled treatments, with five of those six patients indicating they would recommend the home-based approach to others. 

ENHERTU® Plus Pertuzumab Granted Breakthrough Therapy Designation in the U.S. as First-Line Therapy for Patients with HER2 Positive Metastatic Breast Cancer

ENHERTU® (fam-trastuzumab deruxtecan-nxki) in combination with pertuzumab has been granted Breakthrough Therapy Designation (BTD) in the U.S. for the first-line treatment of adult patients with unresectable or metastatic HER2 positive breast cancer. ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Basking Ridge-based Daiichi Sankyo and is being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. The U.S. Food and Drug Administration (FDA) BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

Cabometyx® Approved in the EU for Previously Treated Advanced Neuroendocrine Tumors

Ipsen, with a site in Basking Ridge, announced that the European Commission has approved Cabometyx® (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues. Most forms of neuroendocrine tumors (NETs) develop slowly, can originate in various parts of the body and require multiple lines of therapy as the disease progresses. Treatment options upon progression are often limited depending on primary tumor site and other factors, making it challenging to define optimal sequencing of treatments specific to individual patient needs. In particular, for the 27% of people diagnosed with lung NETs, there have been no approved treatment options upon progression on a prior therapy.

N.J. Named National Leader in AI Innovation

Nonprofit civic tech leader Code for America named New Jersey as one of only three states achieving the top-level designation of “Advanced” AI readiness. The designation underscores New Jersey’s leadership in AI and reflects sophisticated capabilities, comprehensive frameworks, and innovative approaches across three key areas related to AI adoption and implementation in state government: Leadership and Governance, AI Capacity Building and Technical Infrastructure and Capabilities. “The report, titled ‘Government AI Landscape Assessment,’ gave the State high marks: “New Jersey is advanced in its strong commitment to AI development through comprehensive policies, ethical frameworks, capacity building and infrastructure investments. The State’s proactive approach positions it as a leader in responsible AI adoption and innovation.” With regard to leadership and governance, the report highlights Gov. Murphy’s establishment of the AI Task Force that has used artificial intelligence to engage with thousands of residents and workers. 

Quest to Offer FDA-Approved Alzheimer’s Blood Test

Secaucus-based BioNJ Member Quest Diagnostics will soon begin offering a new blood-based in-vitro diagnostic test for Alzheimer’s. The disease affects nearly 7 million Americans. The Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio IVD test from Fujirebio is the first blood-based IVD test approved by the U.S. Food and Drug Administration to aid in identifying patients with amyloid pathology associated with Alzheimer’s. It is designed to aid the clinical assessment of Alzheimer’s in adults age 50 and older presenting at a specialized care setting with signs and symptoms of cognitive decline. The new Fujirebio test builds on Quest’s portfolio of diagnostic tools for assessing patients with cognitive impairment for Alzheimer’s. Quest added that about 12% to 18% of adults over age 60 are living with mild cognitive impairment, a potential sign of Alzheimer’s.

Johnson & Johnson Launches VIRTUGUIDE™ AI-Powered Patient-Matched Lapidus System in U.S. to Reduce Complexity in Bunion Surgery for Millions

New Brunswick-based Johnson & Johnson MedTech announced the launch of the VIRTUGUIDE™ System. This AI-powered, patient-matched solution is designed to support Lapidus procedures, a type of bunion surgery that helps realign the foot by joining two bones near the arch (the first metatarsal bone and the medial cuneiform). The system uses pre-operative planning software, developed in collaboration with PeekMed^®, to assess each patient’s bunion and make personalized recommendations for the intended correction. Bunions are among the most common foot problems, affecting nearly one-third of adults in the U.S., yet traditional Lapidus surgery to treat the condition is often challenging. The VIRTUGUIDE™ AI Lapidus System addresses this by enabling a streamlined approach and reducing surgical complexity. Early users estimated procedural time savings of at least 30 minutes when using the system compared to their previous technique.

BD’s Biosciences & Diagnostics Unit to Merge With Waters in Deal Valued at $17.5B

The Biosciences & Diagnostic Solutions business of Franklin Lakes-based BD (Becton, Dickinson & Co.) will be merged with Waters in a deal valued at $17.5 billion. Upon closing of the transaction, the combined companies will be a powerhouse in liquid chromatography, mass spectrometry, flow cytometry and diagnostic solutions. The combined company will continue to operate under the Waters name and retain its listing on the New York Stock Exchange. “We are bringing together complementary portfolios and channels that create an industry-leading life sciences and diagnostics company. We see an incredible opportunity to leverage both companies’ commitments to unparalleled innovation, technology, and commercial presence to serve attractive high-growth end-markets, while simultaneously unlocking multiple new growth vectors,” Tom Polen, Chairman, CEO and President, BD, said.“

Medtech Research: EDA, Rowan, Cooper to Create State’s 12th Strategic Innovation Center

The N.J. Economic Development Authority and Rowan University will create the state’s 12th Strategic Innovation Center — a multi-location center in Camden and Mullica Hill that will support the research, development and commercialization of novel medical technologies and devices. The SIC will aim to serve as an accelerator for South Jersey’s burgeoning medtech ecosystem. The SIC will serve as a world-class accelerator for early stage companies and promoting overall scientific collaboration in the region. Cooper University Health Care will serve as the anchor commercial affiliate and Garden State Venture Partners will invest in select participating accelerator companies. The SIC will support early stage ideas emerging from southern New Jersey labs and pull companies from across the region into the State.

Lincoln Tech Brings Advanced Manufacturing Training Back to Mahwah Campus

Lincoln Educational Services Corporation has announced the return of career training for manufacturing and machining at its campus in Mahwah. Starting in September, students will be able to earn a Certificate in Advanced Manufacturing with Robotics, working on equipment provided by global leader Haas Automation. The field is projected to add hundreds of jobs statewide in the next 10 years, according to the U.S. Department of Labor, with qualified technicians available for just a fraction of that number. The return of Lincoln Tech’s Manufacturing program at the Mahwah campus was spurred by demand both from employers seeking job candidates and from students eager to explore a tech-driven in-demand career path. Lincoln Tech’s program trains future Advanced Manufacturing professionals with the skills to program, operate, and maintain computerized (CNC) milling and turning machines. Students also learn to read and interpret blueprints, perform precision measurements and operate robotic systems used in automated manufacturing environments.

Biomedical Engineer at Rowan Awarded $650K to Advance Nanoparticle Research in Maternal Health

Rachel Riley, Ph.D., an assistant professor in the Department of Biomedical Engineering in the Henry M. Rowan College of Engineering, is applying her expertise in lipid nanoparticles (LNPs)—tiny, fat-based particles that can deliver therapeutics and genetic material to specific tissues — to better understand and treat pregnancy-related diseases such as preeclampsia. With a five-year, $650,000 National Science Foundation (NSF) Faculty Early Career Development (CAREER) Award, Riley’s IMPACT Lab is exploring new ways to study and treat preeclampsia and other pregnancy-related conditions using nanoparticles. The award enables Riley’s team to investigate placental development and its effects on maternal and fetal health, with the goal of transforming how these diseases are studied and treated. Students in Riley’s lab will engage in both the design and biological aspects of the research. 

EDA Providing Support for Innovation Business Hub of Hispanic Chamber at NJCU 

In a strong show of support for fostering entrepreneurship, workforce development and community engagement initiatives in the Hispanic community, the N.J. Economic Development Authority committed $75,000 to support expansion of the Innovation Business Hub at New Jersey City University that is being run by the Statewide Hispanic Chamber of Commerce. The Innovation Business Hub offers a physical location for local business owners and leaders to gather and attend workshops, seminars and networking events aimed to strengthen connectivity, provide educational resources and tools, foster innovation and spur economic growth. The Hub will offer comprehensive technical education programs, providing small businesses with access to an educational platform where they can take courses, review materials and access key resources. 

First Lady Murphy & Gov. Murphy Break Ground on Trenton’s Maternal and Infant Health Innovation Center

First lady Tammy Murphy and Governor Phil Murphy joined representatives from the New Jersey Economic Development Authority (NJEDA) and the New Jersey Maternal and Infant Health Innovation Authority (MIHIA); state and local government officials; and health care, community and faith leaders to break ground on the first-of-its-kind Maternal and Infant Health Innovation Center (MIHIC) in Trenton. The MIHIC will serve as the epicenter for advancing equitable maternal and infant health care across Trenton and the nation. As a beacon of hope and innovation, it will lead efforts to deliver excellence in perinatal and family care, cultivate a diverse and skilled workforce, conduct cutting-edge policy research, provide integrated wraparound supports and operate a robust data collaborative to better serve families across the State.

NJEDA Closes on Two New NJ Innovation Evergreen Fund Investments

The New Jersey Economic Development Authority (NJEDA) recently closed on two new investments through the New Jersey Innovation Evergreen Fund (NJIEF). Nascent Materials, Inc. received an initial Qualified Investment of $750,000 through an application for co-investment submitted by the Princeton-based venture capital firm, SOSV. Additionally, Enquyst Technologies, Inc. received an initial Qualified Investment of $3 million through an application for co-investment submitted by the New York City-based venture capital firm Eckuity Capital. The NJIEF, launched in 2022, is a unique tool to increase access to strategic resources and venture capital in New Jersey. Under the NJIEF, the State acts as an equity investor in early-stage companies, deploying up to $600 million into companies alongside professional venture capital firms. The Evergreen Fund currently has approximately $19 million of unallocated capital available to fund additional investments into high-growth businesses in New Jersey.

NJEDA to Auction $60M in Tax Credits to Support Food Desert Relief Efforts and Innovation Evergreen

The New Jersey Economic Development Authority is opening applications for the Food Desert Relief Tax Credit Auction and the New Jersey Innovation Evergreen Fund Tax Credit Auction. Up to $10 million in tax credits will be up for sale through the Food Desert Relief Tax Credit Auction and proceeds will fund programs that advance the priorities established by the Food Desert Relief Act. The New Jersey Innovation Evergreen Fund Tax Credit Auction will sell up to $50 million in tax credits to fuel the NJIEF. Under the NJIEF, the State will become an equity investor in startups deploying up to $600 million into companies alongside professional venture capital firms. Applications for both auctions will open on September 2, 2025, at 10:00 a.m. and close on October 3, 2025, at 5:00 p.m.

NJ is Making Largest Manufacturing Investment in State History

The New Jersey Legislature recently passed a bill that creates the Next New Jersey Manufacturing Program, which will provide $500 million in new tax credits for manufacturing, marking the largest manufacturing investment in state history. “We’re giving manufacturers and entrepreneurs the tools they need to grow, compete globally, and succeed right here in the Garden State,” said bill co-sponsor Senator Michael Testa (R-1), at a manufacturing webinar hosted by NJBIA yesterday. “It is a modern revival of the message proudly displayed on the Lower Trenton Bridge — ‘Trenton Makes, The World Takes.’” The program will incentivize various manufacturing activities, including advanced manufacturing, food and beverage production, life sciences, defense, and clean energy component manufacturing, all with the aim to drive innovation, create jobs, and bolster the overall manufacturing sector in the State.

Dr. Massacesi Joins Bristol Myers Squibb in Multiple Roles

Princeton-based BioNJ Member Bristol Myers Squibb announced the appointment of Cristian Massacesi, M.D., as Executive Vice President, Chief Medical Officer and Head of Development. In this role, Dr. Massacesi will oversee the company’s early stage and late-stage product development across all therapeutic areas. Dr. Massacesi began his career in the health care field as a medical oncologist, bringing the ethos of putting patients first into his more than 20 years of global biopharmaceutical experience. He has a proven track record of advancing breakthrough science, from early through late-stage development and global regulatory approvals, inspiring and encouraging his teams to identify novel strategies and take smart risks. He most recently served as Chief Medical Officer of AstraZeneca and Alexion, and as Oncology Chief Development Officer at AstraZeneca, where he led a global organization of more than 3,000 employees.

Maziar Mike Doustdar Appointed as President and Chief Executive Officer of Novo Nordisk

Plainsboro-based BioNJ Member Novo Nordisk announced that Maziar Mike Doustdar has been appointed as president and Chief Executive Officer, effective 7 August 2025. Mike Doustdar succeeds Lars Fruergaard Jørgensen, who will step down as president and chief executive officer on the same date. The company also announced other executive-level changes, effective 7 August. Mike Doustdar, currently Novo Nordisk’s Executive Vice President of International Operations, has a strong track record of creating value and driving growth. Under his leadership over the past decade, Novo Nordisk’s International Operations have more than doubled sales to approximately DKK 112 billion in 2024. The business includes all of Novo Nordisk’s affiliates outside of the US and employs nearly 20,000 people.

Kenvue Announces CEO Transition; Appoints Kirk Perry as Interim CEO

Summit-based Kenvue announced its Board of Directors has made a Chief Executive Officer transition and Kirk Perry has been named as Interim CEO. Current Director and proven CPG and technology leader, Mr. Perry has more than 30 years of global consumer-packaged goods, technology and business transformation experience and a proven track record. He most recently served as President and Chief Executive Officer of Circana, a global provider of technology, data and predictive analytics for the consumer, retail and media sectors, and he continues to serve on the Circana board. Previously, Mr. Perry spent 23 years with Procter & Gamble, where he held several positions of increasing responsibility in general management and marketing roles.

Aquestive Therapeutics Names Sherry Korczynski as Chief Commercial Officer

Warren-based Aquestive Therapeutics announced Sherry Korczynski has been elevated from Senior Vice President of Sales and Marketing to Chief Commercial Officer. “Sherry’s promotion reflects her exceptional track record in commercial leadership within the allergy market,” Daniel Barber, President and CEO of Aquestive said. “Her proven expertise — including her strategic oversight of the EpiPen® brand at Mylan—makes her uniquely qualified to drive our global commercial strategy for Anaphylm. With her deep market knowledge and demonstrated success in marketing, advocacy, public relations and sales execution, Sherry is perfectly positioned to lead our efforts as we bring Anaphylm to patients in the U.S. and abroad.” Ms. Korczynski joined Aquestive in February 2024 to lead the company’s sales and marketing initiatives. 

Vascarta Adds PhRMA/Biotech Veteran Janet Loesberg to Leadership Team

Pharmaceutical firm Vascarta announced that Dr. Janet Loesberg has joined the Summit-based firm as Fractional Chief Strategy Officer. Ms. Loesberg will be responsible for corporate strategy development, portfolio and pipeline management, strategic communications and fundraising/investor relations. She will report to Dr. Richard Prince, chairman, CEO and President. Ms. Loesberg brings a wealth of pharmaceutical and leadership expertise to Vascarta. She has served in executive global medical affairs and R&D roles at Blueprint Medicines, Janssen Pharmaceuticals (div. of Johnson & Johnson), Bristol-Myers Squibb, and Pfizer. She was a board member at Ambrx, a clinical stage biotech company. Most recently, as Senior Vice President of Blueprint Medicines, she was responsible for the medical strategy behind the successful launch of Ayvakit™ (avapritinib) for indolent systemic mastocytosis, a rare hematologic disorder.

Eisai Listed for 24th Consecutive Year in FTSE4Good Index Series, an Index for Socially Responsible Investment

BioNJ Member Eisai Co., Ltd. announced that it has been included in the FTSE4Good Index Series for the 24th consecutive year since its initial inclusion in 2002. The FTSE4Good Index Series is a global index series for socially responsible investment. The FTSE4Good Index Series was developed by FTSE Russell to promote investment in companies that meet global standards for Environmental, Social and Governance (ESG). Eisai’s corporate concept is to give first thought to patients and people in the daily living domain, and to increase the benefits that health care provides to them, as well as address diverse healthcare needs worldwide. By strengthening its ESG initiatives and increasing non-financial value, Eisai is striving to sustainably enhance corporate value based on this concept.

Unleash the Potential of Your Cancer Technology with NCI SBIR Support

July 15 – August 14 | Hosted by National Cancer Institute

Are you working on a cancer technology innovation? Join NCI SBIR’s virtual event series, Unleash the Potential of Your Cancer Technology, taking place July 15–August 14, 2025. This regional webinar series is designed for biotech innovators across the 10 federally recognized U.S. regions and will highlight NCI SBIR funding opportunities, commercialization resources, application tips, and success stories from entrepreneurs in your area. Learn how to access national and regional support to move your cancer technology from lab to clinic.

Doing Business in Eurasia

Throughout Spring 2025 - Fall 2026 | Hosted by Mid-Atlantic - Eurasia Business Council

Mid-Atlantic - Eurasia Business Council is pleased to invite you to its Doing Business in Eurasia seminar series that will be taking place throughout Spring 2025 - Fall 2026. The upcoming seminars organized by the Mid-Atlantic - Eurasia Business Council will be held in Philadelphia, PA; Harrisburg, PA; Wilmington, DE; Pittsburgh, PA; New York, NY; Allentown, PA; Baltimore, MD; and Princeton, NJ. The Doing Business in Eurasia seminar series addresses emerging business opportunities for foreign companies and discusses the legal and regulatory environment in Eurasian countries, including Central Asia, Eastern Europe and European Union.

Students 2 Science STEM Volunteer Opportunities

Inspire the next generation of STEM leaders – Volunteer at Students 2 Science (S2S), a recognized leader in providing rigorous, hands-on STEM experiences for students in 5th-12th grades. S2S is currently recruiting volunteers for its in-lab ISAAC program, which offers rigorous, hands-on STEM experiences for middle and high school students in state-of-the-art laboratories in East Hanover and Newark, NJ. For more information, click here. To register to volunteer with S2S or if you have any questions, please email info@students2science.org or call (973) 947-4880 ext. 545.