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QurAlis is developing precision therapeutics for ALS, a terminal disease that causes muscle paralysis through degeneration of the motor system. We are digging deep into the root causes of the multiple sub-forms of this destructive disease and focus our programs on tackling specific disease-causing mechanisms.
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.
Boston Immune Technologies & Therapeutics based Inc
Repare Therapeutics is developing new, precision oncology drugs for patients that target specific vulnerabilities of tumor cells. Its approach integrates insights from several fields of cell biology including DNA repair and synthetic lethality. Repare`s platform combines a proprietary, high throughput, CRISPR‐enabled gene editing target discovery method with high‐resolution protein crystallography, computational biology and clinical informatics.
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