Alternatives to Rocket Pharmaceuticals

A global biotechnology company focused on developing and commercializing therapies for rare genetic diseases.

Clinical-stage genetic medicine company developing AAV-mediated treatments for cardiovascular and Alzheimer's diseases.

Biotechnology company developing immuno-neurology therapies for neurodegenerative diseases.

Gene therapy company developing treatments for rare inherited diseases.

A biopharmaceutical company focused on developing novel therapies.

Clinical-stage biopharmaceutical company developing gene therapies for rare genetic diseases.

Biotechnology company developing gene therapies and regenerative medicine for heart disease.

Gene therapy company developing transformative treatments for severe genetic diseases.

Biopharmaceutical company focused on rare genetic diseases of obesity.

Ophthalmic pharmaceutical company developing novel treatments for ocular diseases.

Biopharmaceutical company developing novel therapies for rare and ultra-rare genetic diseases.

A global pharmaceutical and biotechnology corporation developing innovative medicines and vaccines for various therapeutic areas.

Biopharmaceutical company focused on developing RNA-targeted therapies for rare genetic diseases.

RD-Biotech develops innovative solutions for drug discovery and development using advanced biotechnology.

Biopharmaceutical company developing therapies for neurodegenerative diseases, focusing on crossing the blood-brain barrier.

Vaxart is a clinical-stage biotechnology company developing oral vaccines for infectious diseases.

Omega Therapeutics is developing a new class of programmable medicines called Epigenomic Controllers.

A biotechnology company developing gene therapy treatments for rare, debilitating genetic diseases.

Moderna is a biotechnology company pioneering mRNA medicine, best known for its COVID-19 vaccine and a broad pipeline of mRNA-based therapeutics.

Clinical-stage biotechnology company advancing molecular glue degrader (MGD) medicines.

Epicrispr Biotechnologies is a biotechnology company developing novel epigenetic therapies, with a lead program targeting Facioscapulohumeral Muscular Dystrophy (FSHD).

A clinical-stage biotechnology company developing inobrodib as a first-in-class, oral p300/CBP inhibitor for the treatment of multiple myeloma.

Developing human-derived antibodies for inflammatory diseases and cancer using AI and proprietary platforms.

Clinical-stage biopharmaceutical company developing microbiome-derived immunotherapies for cancer and immune diseases.

Developing first-and best-in-class antibody-drug conjugates (ADCs) for cancers with high unmet medical need.

A global leader in clinical pharmacology and bioanalytical sciences.

Biopharmaceutical company developing novel small-molecule therapeutics for rare kidney diseases, particularly Autosomal Dominant Polycystic Kidney Disease (ADPKD).

A clinical-stage biopharmaceutical company developing novel therapies for neurological disorders.

A clinical-stage biopharmaceutical company developing novel immunotherapies.

Developing gene editing therapies to lower cholesterol.

Clinical-stage biopharmaceutical company developing novel treatments for exocrine pancreatic insufficiency.

A biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of pulmonary arterial hypertension.

Engineering scalable gene therapies for broader patient populations.

Clinical-stage biopharmaceutical company developing next-generation therapies for obesity and cardiometabolic diseases.

The first Integrated Development and Manufacturing Organization (IDMO) for cell therapies.

Advancing deupirfenidone as a potential new standard of care for Idiopathic Pulmonary Fibrosis (IPF).

Pioneering vision restoration therapies for severe retinal degenerations.

Developing ultrasound-mediated nonviral genetic medicines.

Developing oral small molecule medicines for immunological and chronic diseases.

A clinical-stage biopharmaceutical company developing novel cell therapies for AL Amyloidosis and other serious diseases.

Developing a new class of anti-IgE therapy for multiple atopic conditions.

Advancing clinical stage pipeline targeting new and major neurology and neuropsychiatric disorders.

A biopharmaceutical company focused on developing and commercializing therapies for rare diseases.

Provides regulated, secure, and high-yield digital asset solutions for institutional and corporate treasuries.

Developing Quantum ADCs against the tumor microenvironment for innovative cancer therapies.

A biotechnology company focused on developing novel enzyme-based therapies for reflux disease.

Immunotherapy company targeting oncology and autoimmune diseases.

Developing first-in-class oral therapy for BK polyomavirus infections.

Developing novel male contraceptives.

A biopharmaceutical company developing RNA interference (RNAi)-based medicines.

Developing microbiome-based therapeutics to address underlying disease biology, particularly in gastrointestinal health.

Clinical-stage biopharmaceutical company developing transformative, best-in-class neuropsychiatric therapies.

Scalable, one-time curative in-vivo gene editing therapies.

Advancing scalable microbiome-based therapeutics for severe and chronic diseases.

Clinical-stage immunotherapy company developing novel treatments for cancer.

Advancing targeted alpha therapies.

A biopharmaceutical company focused on drug discovery and development.

A commercial-stage immunotherapy company developing novel treatments for cancer and infectious diseases.

Biopharmaceutical company developing innovative therapies for sepsis and intensive care.