New Jersey’s life sciences community is celebrating another year of groundbreaking medical advancements from the scores of pharmaceutical companies headquartered across the state.

As part of BioNJ‘s 32nd annual dinner meeting and innovation celebration, the industry association recognized those driving discovery. Held Feb. 6 at the Hilton East Brunswick, the evening brought together biopharma leaders, patient advocacy champions, heads of research institutions, and organizations supporting research & development.

In welcoming attendees, Debbie Hart, president and CEO of BioNJ, praised the “passion and dedication to create a brighter, healthier future for countless lives” from New Jersey’s many “trailblazers, visionaries and tireless champions of health and science” who are “continuing to set the standard for excellence nationwide.”

Reflecting on the past 12 months, Hart mentioned several high points, like advancements in the cell and gene therapy field, AI-driven drug development and the advancement of legislation to expand access to biomarker testing for New Jersey patients.

She also highlighted initiatives, such as the first-ever BioNJ Gives Back event in December 2024. Hosted by members Insmed and Genmab, volunteers created 200 hand-stuffed teddy bears and superhero capes as a donation to pediatric patients at Children’s Specialized Hospital.

Additionally, Hart reported progress on BioNJ’s initiative to improve access to clinical trials for its members’ employees. Since issuing a call to action in November 2024, six of the association’s biggest members – Amicus Therapeutics, Bristol Myers Squibb, Genmab, PsychoGenics, PTC Therapeutics and Sanofi – have committed to giving their employees paid time off to take part in clinical trials.

Hart – who sees it as part of BioNJ’s larger mission to advance health equity – thanked the companies for setting a “powerful example” and expressed hope that others will follow suit.

A ‘remarkable year’

As the largest life sciences association in the state, BioNJ represents over 400 research-based organizations across the health care ecosystem, from the largest biopharmaceutical companies to early-stage startups. In addition to advocacy work in Trenton and Washington, D.C., for both the industry and patients, the Trenton-based organization focuses on providing educational events and offering resources to its members.

Dr. Emer Leahy, BioNJ board chair, said, “Looking back, 2024 was an extraordinary year for the industry. Despite its challenges amid numerous financial and regulatory headwinds and a looming patent cliff, we still witnessed groundbreaking scientific advancements, many of which emerged right here in our own backyard.”

Leahy, who is president and CEO of Paramus-headquartered PsychoGenics, a preclinical contract research organization focused on drug discovery and development, said, “Last year was also a remarkable year for BioNJ. The team delivered an astonishing array of initiatives, from hosting 58 programs bringing together more than 5,000 industry professionals to meeting one-on-one with New Jersey policymakers and providing testimony on important legislation.”

“BioNJ’s Value of Medical Innovation campaign remains central to its mission, driving impactful initiatives to educate and inspire change on critical and pressing issues, including the Inflation Reduction Act, PBMs [pharmacy benefits managers] and 340B [drug pricing program]. Plus – a groundbreaking effort to support the recruitment of underrepresented communities in clinical trials – BioNJ and Medidata published a whitepaper, Health Equity in Clinical Trials: New Jersey Participant Demographics,” she said.

Known as the medicine chest of the world, New Jersey is a global leader in discovering and developing new medicines, therapies, diagnostics and technologies to improve the quality of patients’ lives. As an innovation hub for life sciences, New Jersey companies make a steady stream of contributions toward advancing human health.

Over the past year, 18 locally based companies secured 21 novel U.S. Food and Drug Administration approvals. To recognize those achievements, BioNJ issued the following Innovator Awards: Ascendis Pharma (Yorvipath); BeiGene (Tevimbra); Bristol Myers Squibb (Cobenfy); Bracco Diagnostics (Iomervu); Citius Pharmaceuticals (Lymphir); Eli Lilly (Ebglyss and Kisunla); Idorsia (Tryvio); Ipsen (Iqirvo); Galderma (Nemluvio); GE HealthCare (Flyrcado); Geron (Rytelo); Gilead (Livdelzi); Johnson & Johnson (Lazcluze); Merck & Co. (Capvaxive and Winrevair); Novo Nordisk (Alhemo); Pfizer (Beqvez and Hympavzi);PTC Therapeutics (Kebilidi); Sun Pharma (Leqselvi).

Young honoree

The nonprofit also presented its 7th annual Heart of BioNJ Awards, which are given to individuals for their dedication and selfless work on behalf of patients.

After introducing Grace Eline and Dr. Husseini Manji as this year’s honorees, Leahy cited BioNJ’s ongoing push to connect with patients and patient advocate and encourage them to share their stories.

“The team brought these voices to the forefront, facilitating meaningful conversations between patients, patient advocates and legislators. They also captured compelling stories in video and worked tirelessly to push for legislation that ensures patients gain better access to the treatments they deserve. These powerful stories of courage, resilience and hope remind us of why our work matters,” she said.

Eline, a 16-year-old brain cancer survivor from Long Hill, is the founder of the WITH Grace Initiative. Launched in partnership with The American Childhood Cancer Organization, the charity is dedicated to pediatric cancer research funding, advocacy for the cause, and direct care initiatives to those fighting the disease and their families.

At just 9 years old, Eline was diagnosed with Germinoma, a germ-cell brain tumor. After that, she traded in her softball glove and gymnastics leotard for treatment of this very rare cancer.

Eline then began four months of intense chemotherapy at Newark Beth Israel followed by 24 rounds of proton radiation at Robert Wood Johnson and Rutgers Cancer Institute of New Jersey.  She was pronounced cancer-free in December 2018.

The Watchung Hills High Student will spend the rest of her life being monitored for recurrence of cancer but currently shows no evidence of disease. She remains passionate about being a voice for kids with cancer and wants to help find a cure. As part of that effort, Eline has helped several charities, as well as formed her own in 2020.

She has also represented the cause at the state, national and international levels.  During President Donald Trump’s 2019 State of the Union address, Eline was one of 13 guests invited to attend as a guest of honor. The president described her as “very brave” and used Eline’s story to encourage Congress to support a $500 million increase in funding for childhood cancer research.

“During my treatment, I met kids who came from all over the country to meet doctors and go to certain hospitals. This made me realize just how lucky I was to receive my treatment only 30 minutes away from home. Although being diagnosed with cancer was horrible, I was extremely lucky to be diagnosed in New Jersey where some of the best hospitals and doctors were and I wouldn’t have been able to get through my journey if it weren’t for my amazing staff of doctors and nurses,” Eline told attendees at BioNJ’s event.

“This December I just celebrated six years cancer free … I was able to start high school, play club sports and even learn how to drive. Since the start of my journey, I have been very lucky to have many opportunities to share my story, such as being at the White House and the Capitol to represent pediatric cancer on a federal level as well. I have worked at the state level to pass one-of-a-kind legislation for research. I’ve also been able to found my own organization called the With Grace initiative, which focuses on giving back against the fight of pediatric cancer through direct care research and overall awareness. Unfortunately, I know that not all kids are as lucky as me and they don’t have a voice, which is why I am here tonight to not only share my story but to give many of those kids a voice,” Eline explained.

“Through my treatment and my advocacy, I’ve been able to understand the importance of research. Many kids like me experience side effects, some of which are long-term and will affect us for the rest of our lives. This is usually because the treatments that we get are not only decades old, but designed for adult bodies which will leave us with a lifetime of harmful side effects,” she said.

“By funding innovative research at the state and the federal levels and ensuring kids are included in clinical trials, we are able to discover new treatments that will not only cure our cancers, but make treatments easier and prevent harmful, long-lasting side effects,” she said. “This innovation can help more kids like me live longer, more fulfilling and happy lives.

‘A visionary leader’

Manji is the former global head of neuroscience at New Brunswick pharma giant Johnson & Johnson, an Oxford University professor and co-chair of the U.K. Government’s Mental Health Mission. Manji is also past global therapeutic head for neuroscience at Janssen Research & Development pharmaceutical companies and global head, Science for Minds, at J&J.

He is also credited with spearheading development of J&J’s ketamine-based antidepressant nasal spray Spravato. Last month, it was approved by the FDA as a standalone therapy for adults with treatment-resistant depression.

Spravato first received authorization in 2019 for use in combination with oral antidepressants in patients struggling with major depressive disorder. At the time, it marked the first new major depression treatment to win FDA approval in more than three decades.

Before joining J&J, Manji was chief of the laboratory of molecular pathophysiology at the National Institutes of Health and director of the NIH Mood and Anxiety Disorders Program, the largest program of its kind in the world.

During his 25-plus year career, Manji has helped discover, develop and launch several new medications for serious neuropsychiatric and neurodegenerative disorders. Besides Spravato, that includes a once every six-month treatment for schizophrenia, novel mechanism(s) for Alzheimer’s Disease and multiple sclerosis among others.

Manji has also been actively involved in developing biomarkers to help refine these multifactorial diseases and to develop a holistic approach toward neuropsychiatric and neurodegenerative disorders.

Leahy described him as “a visionary leader known for his innovative discoveries for patients suffering from severely disabling mental illnesses,” saying, “His work has led to transformative advances in neuroscience, including Alzheimer’s disease and pioneering treatments for depression and schizophrenia.”

Hart praised Manji for his dedication to “bringing leaders from research policy investment in healthcare together to drive innovation and improve patient care globally.”

“And as a mentor and thought leader, he inspires the next generation of researchers to push boundaries and foster bold advancements for his contributions to mental health research and improved patient outcomes,” she said.

In accepting the award, Manji said, “It’s humbling and a true honor … and a testament to what we can collectively achieve when science, collaboration and purpose converge.”

“I really believe mental illness represents one of the most challenging health, economic and social justice issues of our time. Its impacts are pervasive, touching individuals, families and societies in profound ways, and yet it has long been overlooked, misunderstood and stigmatized,” he said. “I’ve always believed, like all of you, in the power of the ability of biomedical research to transform lives — whether it’s understanding the molecular mechanisms behind disease pathology or developing treatments that can save lives. It is truly a field that impacts humanity in profound ways. I’ve witnessed firsthand the transformative power of innovation.

“I think we all know that the journey to discovery is not linear, and there’s lots of challenges along the way, but recent advances in neuroscience and mental health demonstrate that, despite the complexity, if we stay the course persevere with scientific rigor and collaborative action, we can be successful,” Manji continued. “But we have to acknowledge that our work is far from done. The stigma of mental illness remains a barrier to care. The inequities in access to treatment are stark, and the global burden of mental illness continues to grow. Addressing these challenges requires not just groundbreaking science, but a collective commitment to ensuring that the fruits of innovation reach all those in need.”

Manji urged the leaders, advocates and innovators at the event to remember “how collectively powerful we can be.”

“We have the power to build a future where mental illness is treated with the same urgency and dignity as any other area of medicine – a future where no one is left behind,” he said.

Redefining the role of patient advocacy

BioNJ also recognized Jayne Gershkowitz with the 17th annual Dr. Sol J. Barer Award for Vision, Innovation and Leadership.

As one of the founding leaders and chief patient advocate at rare disease company Amicus Therapeutics, Gershkowitz’s passion for fostering meaningful partnerships between the patient community and biopharmaceutical industry has set a gold standard in orphan drug development. Through her leadership, she has redefined the role of patient advocacy shaping it into one of strategic impact and transformative influence.

Barer, who was on hand to present his namesake award, said, “With an innovative mindset and unwavering patient-centric philosophy, our honoree has elevated the role of patient advocacy within the biopharma industry … Gershkowitz has devoted her career to ensuring that patients and their families are not only heard, but also actively engaged at every stage of the drug development process.

“Jayne was instrumental in embedding the needs of individuals living with rare diseases into Amicus’ global strategies and daily operations, helping shape its mission and impact,” said Barer, former chair and CEO of Celgene Corp. and current Teva Pharmaceutical Industries chair. “It has often been said that ‘Innovation requires collaboration, vision and the courage to challenge the status quo.’  Jayne personifies each of these qualities in their truest form.”

The evening also featured a fireside chat with John Crowley, president and CEO of global trade association Biotechnology Innovation Organization, and Dr. Peter Marks, director of the center for biologics evaluation and research at the FDA.

BioNJ also kept the tradition of a surprise performance, with this year’s event kicking off with a fresh act from Smash Entertainment. New to program: a donation on behalf of attendees to UNICEF that totaled nearly 6,000 vaccines to babies around the world.