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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.
Stemline Therapeutics is a clinical stage biopharmaceutical company developing oncology compounds that target cancer stem cells.
At Inipharm, we`re building on novel insights and applying our chemistry expertise to develop therapies for patients with liver disease. Inipharm`s lead program is focused on small-molecules to target the activity of HSD17B13, a highly validated, genetically defined target for multiple severe liver and related diseases.
Rapid, Personalized T-cell Therapies for Cancer on BlueSphere Bio…
We operate with a “No Analog” mindset—continually pushing boundaries, navigating new terrain and unleashing fresh thinking. We are committed to seeking better outcomes which compels us to ask questions that have not been asked before or remain unresolved. We are driven by a desire to create meaningful impact across business, health and society to ease the burden on people living with chronic diseases. Intarcia is a rapidly emerging biotech company dedicated to redesigning the treatment, prevention and experience of chronic disease. Our cross-disciplinary teams include forward-thinking scientists and entrepreneurs collaborating in a fast-paced environment redefining the future of medicine.