DFNB16, a recessive form of mild-to-moderate human deafness, is caused by mutations in the STRC gene, which encodes stereocilin, a protein essential for the effective function of outer hair cells as cochlear amplifiers. Researchers from the Institut Pasteur and Sorbonne Université in Paris, France, have developed and evaluated the efficacy of an Strc gene therapy in a mouse model of DFNB16.
Seamless Therapeutics has received big pharma endorsement of its proprietary recombinase gene editing platform, sealing a potential $1.12 billion deal with Eli Lilly and Co. to apply the technology in hearing loss.
Siren Biotechnology Inc. has obtained IND approval from the FDA enabling the initiation of its first-in-human trial for its lead investigational program SRN-101 in adult patients with recurrent high-grade glioma.
Sensorion SA has announced a €60 million (US$72 million) financing, with a €20 million (US$24 million) strategic investment from Sanofi SA, as it advances its pipeline of therapies to restore, treat and prevent hearing loss disorders.
Scientists from the Smidt Heart Institute, Cedars-Sinai Medical Center (Los Angeles, CA, USA) recently explored adeno-associated virus (AAV) vectors carrying the T-box transcription factor 18 (TBX18) transgene to produce a physiologically responsive biological pacemaker activity for the treatment of bradycardia.
Adolore Biotherapeutics Inc. has announced that the FDA has granted orphan drug designation to the company’s Kv7-activating rdHSV-CA8* gene therapy for treatment of primary and secondary erythromelalgia.
Généthon, a nonprofit laboratory created by the AFM-Téléthon, has reported its exclusive, worldwide licensing agreement with Askbio Inc., a subsidiary of Bayer AG, for the use of a patented component of AB-1009, under development by Askbio, for the treatment of Pompe disease.
The U.S. FDA has cleared Tessera Therapeutics Inc.’s IND application for TSRA-196, its lead in vivo gene editing therapy for α-1 antitrypsin deficiency (AATD). The Australian Human Research Ethics Committee (HREC) has also granted approval for the company to begin the phase I/II clinical study.
Askbio Inc., a subsidiary of Bayer AG, has received IND clearance from the FDA for AB-1009, an AAV gene therapy being developed for the treatment of late-onset Pompe disease.
Genprex Inc. has released preliminary in vivo proof-of-concept data for GPX-002, the company’s diabetes gene therapy drug candidate, in a nonhuman primate (NHP) model of type 2 diabetes and in a mouse model of type 2 diabetes. GPX-002 is being developed for the potential long-term control of both type 1 and type 2 diabetes.
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