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GenSight Biologics (IPO) is in the final registration phase of what could be the 2nd gene therapy ever approved for an ophthalmic condition. In addition, GenSight has been the 1st company to ever perform an optogenetics technology-based clinical trial in humans.
Quethera was acquired by Astellas (Takeda) for its clinical pipeline to treat glaucoma using Gene Therapy technology, which also has potential applications to treat CNS diseases such as Alzheimer’s and Parkinson’s. Takeda will be starting first-in-human clinical trials for glaucoma soon and is exploring the therapy for neurodegenerative diseases.
Orchard Therapeutics (IPO) development programs focus on restoring normal gene function in primary immune deficiencies and metabolic diseases. Orchard’s pioneering technology uses a sample of the patient’s own stem cells, which are modified with a functioning copy of the missing or faulty gene before being transplanted back into the patient’s body.