From Boston to Philadelphia and throughout the Northeast of the United States, the East Coast remains one of the most dynamic regions in the world for life sciences innovation. As TFS HealthScience prepares to attend the 2025 Outsourcing in Clinical Trials (OCT) East Coast conference, we’re spotlighting ten of the most promising emerging biotech and specialty pharmaceutical companies shaping the future of clinical research.

The East Coast Biotech Corridor: A Thriving Innovation Ecosystem

The East Coast is home to one of the world’s most powerful life sciences corridors — stretching from Boston’s biotech epicenter through New York and Philadelphia and down to North Carolina’s Research Triangle Park (RTP). This region fosters a uniquely rich environment for innovation, with its proximity to elite academic institutions, high investment volume, and deeply collaborative culture across startups, contract research organizations (CROs), and global pharma leaders.

• Boston/Cambridge, Massachusetts is widely regarded as the global biotech capital, with over 1,200 life sciences companies and some of the world’s most advanced R&D pipelines.

• Philadelphia, Pennsylvania continues to rise as a top biotech hub, home to more than 1,600 life sciences companies and strong gene and cell therapy infrastructure.

• Research Triangle Park, North Carolina boasts a deep bench of biotech, pharma, and CRO talent, with major companies like Biogen, GSK, and Eli Lilly contributing to its growth.

With hubs like these, the East Coast offers fertile ground for early-stage companies to access capital, talent, and experienced partners — making it one of the most exciting regions to watch in 2025.

Whether presenting at the upcoming event or quietly advancing transformative science behind the scenes, these companies reflect the agility, vision, and therapeutic focus that define the next generation of sponsors. We’re proud to recognize their work — and excited about the opportunities to collaborate.

1. Marinus Pharmaceuticals

Marinus Pharmaceuticals is a clinical-stage pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, particularly in rare and refractory epilepsy. Best known for its work in CDKL5 deficiency disorder (CDD), a severe and rare genetic epilepsy — Marinus developed and commercialized ZTALMY® (ganaxolone), the first FDA-approved therapy specifically indicated to treat seizures associated with CDD in patients two years of age and older. The approval was supported by results from the pivotal Phase 3 Marigold Study.

Beyond CDD, Marinus has been advancing ganaxolone across other indications. Its pipeline includes intravenous ganacolone for refractory status epilepticus (RAISE trial) and oral ganaxolone for tuberous sclerosis complex (TrustTSC trial).

In February 2025, Immedica Pharma AB completed its acquisition of Marinus, making the company a wholly owned subsidiary focused on expanding rare disease programs across global markets. Together with Immedica, Marinus is poised to expand access to novel treatments for patients with serious seizure disorders.

2. Aro Biotherapeutics

Aro Biotherapeutics is a clinical-stage biotechnology company pioneering the development of potent, tissue-targeted genetic medicines through its proprietary Centyrin platform. By reimagining targeted drug delivery, Aro aims to enhance the activity of genetic medicines in extra-hepatic tissues—enabling tissue-specific delivery of RNA therapeutics to cells that traditional delivery systems often fail to reach.

The company’s lead candidate, ABX1100, is being developed for Pompe Disease, a rare neuromuscular disorder. In early 2025, Aro dosed the first patient in a Phase 1b trial of ABX1100 following FDA clearance of its investigational new drug application. The program holds both Orphan Drug and Rare Pediatric Disease designations.

In addition to Pompe disease, Aro is advancing new Centyrin-siRNA programs targeting autoimmune and inflammatory conditions. The company raised $41.5 million in Series B financing in late 2023 to support pipeline expansion and, in early 2025, underwent a leadership transition as co-founder Susan Dillon, Ph.D., became Board Chair and Purnanand Sarma, Ph.D., was appointed CEO. With a differentiated platform and experienced leadership, Aro is positioned to accelerate the delivery of targeted RNA therapies for patients with serious and underserved diseases.

3. Castle Creek Biosciences

Castle Creek Biosciences is a late-stage biotechnology company advancing innovative gene therapies for rare dermatologic and metabolic diseases. The company’s lead candidate, D-Fi (dabocemagene autoficel), is an ex vivo autologous fibroblast gene therapy currently in a Phase 3 clinical trial for Recessive Dystrophic Epidermolysis Bullosa (DEB), a severe genetic skin disorder. In early 2025, Castle Creek secured $75 million in royalty financing led by Ligand Pharmaceuticals to support the ongoing trial.

Beyond dermatology, Castle Creek is expanding into metabolic liver diseases through its in vivo gene therapy platform, initiated with the acquisition of Novavita Thera in 2022. The company is developing LV-FAH, a lentiviral vector-based therapy for hereditary tyrosinemia type 1 (HT1), a rare and life-threatening metabolic disorder. With late-stage programs and expansion into new therapeutic areas, Castle Creek is advancing a versatile pipeline aimed at transforming care for patients with serious rare diseases.

Discover the latest insights and future prospects in Epidermolysis Bullosa (EB) treatment from TFS, including gene therapy and regenerative approaches. Download our white paper. 

4. Neurogene

Neurogene is a clinical-stage biotechnology company developing gene therapies for rare neurological diseases. The company’s lead candidate, NGN-401, is an investigational AAV9 gene therapy for Rett Syndrome that incorporates Neurogene’s proprietary EXACT™ gene regulation technology. In 2024, NGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation and was selected for the FDA’s START Pilot Program, both aimed at expediting development for promising rare disease treatments.

The company is also evaluating strategic options for NGN-101, its gene therapy program for CLN5 Batten Disease, after discontinuing further development in 2024. With a focus on scalable manufacturing and strategic partnerships, Neurogene is building toward future commercialization of next-generation gene therapies.

5. Azitra

Azitra is a clinical-stage biotechnology company applying synthetic biology and microbiome engineering to advance precision dermatology. The company is developing live biotherapeutic products (LBPs) and protein therapeutics designed to restore critical skin barrier proteins in rare and underserved dermatological conditions.

Azitra’s lead candidate, ATR-12, is an engineered strain of Staphylococcus epidermidis that secretes the LEKTI protein, targeting Netherton syndrome, a rare genetic skin disorder marked by protease dysregulation. In late 2024, the company dosed the first patient in a Phase 1b trial of ATR-12 in adults with Netherton syndrome. Additional pipeline assets include ATR-04, a microbial therapy targeting EGFR inhibitor-induced rash in cancer patients, which entered clinical development following FDA IND clearance in August 2024, and ATR-01, a recombinant filaggrin-secreting therapy for ichthyosis vulgaris, backed by newly issued U.S. patents.

With a platform rooted in skin biology and live therapeutic innovation, Azitra is advancing a new generation of microbiome-based solutions for chronic and rare skin diseases.

6. EvolveImmune Therapeutics

EvolveImmune Therapeutics is advancing the next generation of T cell engager immunotherapies for solid tumors through its proprietary EVOLVE platform. Designed to overcome tumor resistance to current immunotherapies, EvolveImmune’s multi-functional antibodies combine tumor targeting with integrated T cell co-stimulation, including through CD2, to drive robust immune responses—even in low-immunogenic (“cold”) tumors.

The company’s lead preclinical programs include EV-104, which targets ULBP2, a protein enriched in basal-squamous cancers, and EV-106, which targets B7-H4 in breast cancer. Both candidates have demonstrated potent anti-tumor activity in patient-derived models and preclinical studies.

In October 2024, EvolveImmune entered a strategic collaboration with AbbVie, receiving $65 million upfront and up to $1.4 billion in milestones to co-develop next-generation T cell engagers for cancer. With strong science and industry validation, EvolveImmune is shaping the future of immuno-oncology through novel, precision-driven T cell therapies.

7. OnCusp Therapeutics

OnCusp Therapeutics is a translational oncology company focused on in-licensing and accelerating the development of high-potential cancer therapeutics from Asia and other global markets. With a model built on scientific rigor and speed, OnCusp advances preclinical programs toward clinical proof-of-concept with operational efficiency and deep oncology expertise.

The company’s lead program, CUSP06, is an antibody-drug conjugate (ADC) targeting Cadherin-6, being developed for platinum-resistant ovarian cancer and other solid tumors. Licensed from China’s Multitude Therapeutics, CUSP06 has shown encouraging anti-tumor activity with a manageable safety profile in early Phase 1 studies. In February 2025, the FDA granted Fast Track designation to support its expedited development.

Backed by an oversubscribed $100 million Series A round, OnCusp is also advancing a pipeline of additional oncology assets. By bridging Eastern innovation with Western clinical execution, OnCusp exemplifies a new generation of globally integrated, fast-moving biotech leaders in oncology.

Connect with OnCusp’s Vice President of Clinical Operations, Priya Marreddy, at OCT East Coast where she will serve as a panelist in the session, “Navigating the Outsourcing Landscape for Emerging Therapy Areas” on May 21 ad 2:15 PM.

8. Larimar Therapeutics

Larimar Therapeutics is focused on developing treatments for complex rare diseases, with a primary emphasis on Friedreich’s Ataxia (FA)—a progressive neurodegenerative disorder. Their lead candidate, nomlabofusp (CTI-1601), is a recombinant fusion protein designed to deliver frataxin, the deficient mitochondrial protein in FA, directly to patient cells.

In May 2024, the FDA lifted the partial clinical hold on nomlabofusp following a review of Phase 2 dose exploration study data, allowing Larimar to escalate dosing in their ongoing open-label extension study. The company plans to discuss Phase 3 strategy with the FDA after completing the dose exploration.

With regulatory momentum and a clear development path, Larimar is advancing toward delivering the first disease-modifying therapy for Friedreich’s Ataxia.

9. Immunocore

Immunocore is a pioneering biotechnology company specializing in T cell receptor (TCR) bispecific immunotherapies. Their proprietary ImmTAC® platform has led to the development of KIMMTRAK® (tebentafusp), the first approved TCR therapeutic, which targets gp100 in metastatic uveal melanoma (mUM).

KIMMTRAK is approved in 38 countries and has been launched in 21 countries globally. Immunocore is also advancing brenetafusp (IMC-F106C), a PRAME-targeting ImmTAC, currently in Phase 3 trials for first-line advanced cutaneous melanoma and Phase 1/2 studies in ovarian and lung cancers. Beyond oncology, the company is applying its ImmTAX platform to infectious diseases, with Phase 1 trials underway for HIV and hepatitis B, and plans to enter clinical testing for autoimmune conditions like type 1 diabetes in the second half of 2025.

With a robust pipeline and global reach, Immunocore continues to lead in TCR-based therapies across oncology, infectious, and autoimmune diseases.

Connect with Immunocore’s Director of Clinical Outsourcing, Melanie Goodwin, at OCT East Coast where she will serve as a panelist in the session, “Ensuring Your FSP Model is a Success: What Do You Need to Know?” on May 21 ad 9:00 AM.

10. Cognition Therapeutics

Cognition Therapeutics is a clinical-stage biopharmaceutical company developing small-molecule therapeutics targeting the sigma-2 receptor complex to treat neurodegenerative diseases. Their lead candidate, zervimesine (CT1812), is an oral therapy designed to prevent synaptic toxicity from misfolded proteins.

In December 2024, the Phase 2 SHIMMER study in patients with dementia with Lewy bodies (DLB) met its primary endpoint of safety and tolerability, showing significant improvements across behavioral, functional, cognitive, and movement measures. Additionally, a biomarker-defined subgroup in the SHINE study for mild-to-moderate Alzheimer’s disease demonstrated a 95% reduction in cognitive decline compared to placebo. Cognition is also evaluating zervimesine in dry age-related macular degeneration through the ongoing MAGNIFY trial.

With promising results across multiple indications, Cognition Therapeutics is poised to advance zervimesine as a potential treatment for a range of neurodegenerative and ophthalmic disorders.

As the life sciences landscape on the East Coast continues to thrive, emerging innovators like these are redefining what’s possible in clinical development. At TFS, we specialize in supporting biotech and specialty pharma companies through every stage of their clinical journey — with deep expertise in oncology, dermatology, neuroscience, and ophthalmology. With our North American hub in the Research Triangle Park (RTP) region of North Carolina, we’re proud to be part of the East Coast ecosystem driving global breakthroughs forward.

Heading to OCT East Coast 2025?
Let’s connect. Schedule a meeting with us to explore how TFS can be the CRO partner that brings your next study to life — on time, on budget, and with the scientific precision your program deserves.