| Name | Title | Contact Details |
|---|---|---|
Justin McCue |
Chief Technology Officer | Profile |
La Jolla Bioengineering Institute is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Prime Medicine was founded to bring the promise of gene editing to patients. We use Prime Editing, a next-generation technology that can “search and replace” to restore normal genetic function almost anywhere in the genome. Prime Editing searches to find the precise place in the genome to edit and replaces the segment of faulty DNA with a correct copy of DNA. A single Prime Editor can correct the individual mutations found across patients, meaning Prime Editing can potentially address more than 90 percent of known disease-causing genetic mutations.
Roar Biomedical is a New York, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Imara Inc. is dedicated to developing novel therapeutics for people living with sickle cell disease and other hemoglobinopathies. Sickle cell disease is a rare, genetic blood disease that causes red blood cells to sickle and become damaged, activating immune cells and blocking blood flow in capillaries, injuring many organs and causing daily pain. Imara is developing IMR-687, a highly selective, potent small molecule inhibitor of phosphodiesterase-9 (PDE9i), to treat patients with sickle cell disease. Imara was launched following an 18-month scientific collaboration between orphan drug accelerator Cydan Development and H. Lundbeck A/S.
We are a biotechnology company creating a new class of targeted gene therapies for debilitating and life-threatening genetic disorders in both central nervous system (CNS) and non-CNS indications. Current gene therapy approaches have shown dramatic efficacy in several rare diseases but are hindered by imprecise targeting. The inability to selectively transduce specific cells and tissues effectively drives administration of higher doses and resulting safety liabilities. We are addressing these concerns with our proprietary adeno-associated virus (AAV) engineering platform that generates capsids optimized to target specific tissues and cells in the disease organ while limiting transduction of tissues and cells not relevant to the target disease. Through our next-generation AAV platform combined with cargo development and state-of-the-art manufacturing, we are tackling monogenetic and sporadic CNS and non-CNS disorders that have previously been challenging to address.