Astellas Pharma Inc. has invested $50 million in Taysha Gene Therapies Inc. in exchange for 15% of the company and exclusive options to in-license Taysha’s lead gene therapy candidates, TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy.
The extraordinary proliferation of different genetic therapeutic modalities in the last decade has not been matched by a commensurate flourishing of delivery technologies. The liver is the natural destination for many carriers when administered systemically. But getting beyond the liver to other organ systems has proven to be a significant challenge. Only a tiny percentage of carriers – even those with targeting moieties – escape first pass metabolism in the liver and reach their target destination. “The key issue is getting out of the liver,” Nessan Bermingham, founder and interim CEO of Liberate Bio, Inc., told BioWorld. “That’s not a trivial problem.”
The volatile gene therapy space is getting a boost with Eli Lilly and Co.’s acquisition of Akouos Inc., which only a month before had received the first IND from the U.S. FDA for an adeno-associated virus (AAV)-based hearing loss treatment. Lilly plans to pay about $610 million for the company to get at AK-OTOF, Akouos’ lead candidate for treating hearing loss due to mutations in the otoferlin gene.
Decibel Therapeutics Inc. has received clearance from the FDA for its IND application to initiate a phase I/II trial of DB-OTO in pediatric patients, potentially in infants younger than 2 years of age.
Researchers from Meiragtx Ltd. presented preclinical data on a new and optimized gene therapy based on a mini ATP7B gene for the potential treatment of Wilson’s disease (WD), a rare (1:30,000) autosomal recessive genetic disease caused by loss-of-function mutations in ATP7B, leading to a pathologically high amount of copper in the liver and brain.
Glaucoma is an eye disease that damages the optic nerve, with the main cause being ocular hypertension due to high resistance to the outflow of aqueous humor.
Bloomsbury Genetic Therapies Ltd. has raised £5 million (US$5.5 million) in a seed round, to take four gene therapy programs based on research carried out by the scientific founders at University College London into clinical development.
After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.
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