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There are approximately 7,000 rare disorders worldwide. Each day more are being discovered. 95% of rare diseases have not one single FDA approved treatment. Glycomine develops therapeutics for diseases which have no treatment options.
Applied Therapeutics is a clinical-stage biopharmaceutical company that uses our novel drug-development approach to create transformative, life-changing treatments for the patients who desperately need them.
Switch Therapeutics is an emerging preclinical stage biotechnology company pioneering a new type of medicine that integrates nucleic acid nanotechnology and RNA interference (RNAi) science with the goal of treating a range of diseases – affecting the central nervous system and systemic indications – with significant unmet needs. Switch`s novel gene knockdown approach is based on technology developed by renowned researchers in the field of RNA from Caltech, Harvard and City of Hope. Based on the scientific discovery of Switch`s co-founders, the company has developed a novel proprietary platform known as CASi (Conditionally Activated siRNAs) that combines advantageous properties of both single and double-stranded RNAs in a single molecule, allowing for cell selective RNAi activity. To date, Switch has raised $52M in funding through Series A, and the company`s South San Francisco based team has continued to grow as its research has advanced.
Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we hope to rapidly translate our treatments from bench to bedside. We have combined our team`s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to quickly and efficiently build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for new cures—to dramatically improve patients` lives.
Gyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness. The company was acquired by Novartis, a leading global medicines company, in February 2022.