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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.
Valitor, Inc. has developed novel methods of modifying therapeutic proteins to achieve substantial control over their pharmacokinetics, target specificity and bioactivity. Our technology platform can be broadly applied to improve the pharmacological properties of many protein drugs that are currently approved or under development. We currently have development pipelines in the fields of dermatology, ophthalmology, orthopedics, and stem cell therapy that highlight the capabilities of our novel drug products.
Topaz Pharmaceuticals LLC is a Horsham, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Absci is the AI-powered synthetic biology company unlocking the potential of proteins as the next generation of therapeutics. We built our Integrated Drug Creation™ Platform to accelerate discovery of novel biotherapeutic drug candidates and generation of the cell lines to manufacture them in a single efficient process. Biotech and pharma innovators partner with us to develop new protein-based drugs, including those that may be impossible to make with other technologies. Our goal is to enable the creation of better medicines by Translating Ideas into Drugs.
RayzeBio is a biotechnology company focused on improving outcomes for people with cancer by harnessing the power of targeted radioisotopes. With a focus on clinically validated solid tumor targets, RayzeBio is developing novel drug conjugates to deliver potent therapeutic radioisotopes such as Actinium-225, an alpha-emitter. The company is backed by a syndicate of sophisticated healthcare investors and was established in 2020