Discover the top 10 emerging East Coast biotech companies set to revolutionize healthcare and drive innovation in 2025.

Introduction

The East Coast of the United States continues to be a powerhouse for life sciences innovation, hosting a vibrant ecosystem of innovative biotech startups. From Boston’s renowned biotech hub to the dynamic advancements in Philadelphia and North Carolina’s Research Triangle Park, these companies are at the forefront of transformative healthcare solutions. In 2025, these ten emerging biotech firms are poised to make significant impacts in clinical research, personalized medicine, and therapeutic advancements.

1. Marinus Pharmaceuticals

Marinus Pharmaceuticals is a clinical-stage company focused on developing innovative therapeutics for seizure disorders, particularly rare and refractory epilepsy. Their flagship product, ZTALMY® (ganaxolone), was the first FDA-approved therapy for seizures associated with CDKL5 deficiency disorder (CDD) in patients two years and older. With the recent acquisition by Immedica Pharma AB, Marinus is expanding its reach to provide novel treatments for serious seizure disorders globally.

2. Aro Biotherapeutics

Aro Biotherapeutics is pioneering tissue-targeted genetic medicines through its proprietary Centyrin platform. Their lead candidate, ABX1100, targets Pompe Disease, a rare neuromuscular disorder. Following FDA clearance for its investigational new drug application, Aro is advancing clinical trials and expanding its pipeline to include treatments for autoimmune and inflammatory conditions, backed by substantial Series B financing and experienced leadership.

3. Castle Creek Biosciences

Castle Creek Biosciences is advancing gene therapies for rare dermatologic and metabolic diseases. Their lead candidate, D-Fi (dabocemagene autoficel), is in Phase 3 trials for Recessive Dystrophic Epidermolysis Bullosa (DEB). With significant financial backing from Ligand Pharmaceuticals, Castle Creek is also expanding into metabolic liver diseases, aiming to transform care for patients with serious rare conditions.

4. Neurogene

Neurogene is dedicated to developing gene therapies for rare neurological diseases. Their lead candidate, NGN-401, is an investigational AAV9 gene therapy for Rett Syndrome, utilizing their proprietary EXACT™ gene regulation technology. With RMAT designation and participation in the FDA’s START Pilot Program, Neurogene is strategically positioned to accelerate the development and commercialization of next-generation gene therapies.

5. Azitra

Azitra specializes in precision dermatology through synthetic biology and microbiome engineering. Their lead candidate, ATR-12, targets Netherton syndrome, a rare genetic skin disorder. Azitra is expanding its pipeline with therapies for cancer-induced rashes and ichthyosis vulgaris, leveraging live biotherapeutic products to restore critical skin barrier proteins in underserved dermatological conditions.

6. EvolveImmune Therapeutics

EvolveImmune Therapeutics is advancing T cell engager immunotherapies for solid tumors with its proprietary EVOLVE platform. Their lead programs, EV-104 and EV-106, demonstrate potent anti-tumor activity in preclinical studies. A strategic collaboration with AbbVie underscores EvolveImmune’s commitment to developing precision-driven immunotherapies that overcome tumor resistance in cancer treatment.

7. OnCusp Therapeutics

OnCusp Therapeutics focuses on in-licensing and accelerating cancer therapeutics from global markets. Their lead program, CUSP06, targets platinum-resistant ovarian cancer and has received FDA Fast Track designation. With substantial Series A funding, OnCusp is bridging Eastern innovation with Western clinical execution, exemplifying the next generation of globally integrated biotech leaders in oncology.

8. Larimar Therapeutics

Larimar Therapeutics is dedicated to treating complex rare diseases, with a primary focus on Friedreich’s Ataxia (FA). Their lead candidate, nomlabofusp (CTI-1601), is a recombinant fusion protein designed to deliver frataxin directly to patient cells. Recent regulatory advancements have paved the way for Phase 3 strategies, positioning Larimar to deliver the first disease-modifying therapy for FA.

9. Immunocore

Immunocore is at the forefront of T cell receptor (TCR) bispecific immunotherapies with their ImmTAC® platform. Their approved therapy, KIMMTRAK® (tebentafusp), targets metastatic uveal melanoma and is available in 38 countries. Immunocore continues to expand its pipeline with therapies for various cancers, infectious diseases, and autoimmune conditions, maintaining its leadership in TCR-based treatments.

10. Cognition Therapeutics

Cognition Therapeutics is developing small-molecule therapeutics targeting the sigma-2 receptor complex to treat neurodegenerative diseases. Their lead candidate, zervimesine (CT1812), has shown promising results in Phase 2 studies for dementia with Lewy bodies and Alzheimer’s disease. Ongoing trials in age-related macular degeneration highlight Cognition’s potential to address a range of neurodegenerative and ophthalmic disorders.

Conclusion

The East Coast remains a vibrant hub for innovative biotech startups, driving significant advancements in healthcare and clinical research. These ten emerging companies exemplify the region’s dynamic ecosystem, characterized by cutting-edge research, strategic partnerships, and a commitment to transforming patient care. As these firms continue to innovate and expand their therapeutic offerings, they are set to revolutionize the biopharmaceutical landscape and improve health outcomes globally.

Are you interested in partnering with leading biopharmaceutical innovators? Visit Bristol Myers Squibb to learn more about our commitment to advancing patient-centric solutions and driving healthcare innovation.