A recent study published in Molecular Therapy by researchers from West China Hospital at Sichuan University and collaborators aimed to investigate the effect of CaMKII gene therapy on retinal ganglion cell (RGC) protection in mouse models of retinal ischemia/reperfusion (I/R) injury and congenital glaucoma.
Friedreich’s ataxia (FA), the most common form of hereditary ataxia, is an autosomal recessive neurodegenerative disorder affecting multiple organ systems, and causing cardiomyopathy, scoliosis, muscle weakness, speech impairment and other systemic issues.
As many had predicted following news of a clinical hold on the gene therapy last month, Regenxbio Inc. disclosed receipt of a complete response letter (CRL) regarding its BLA for RGX-121 (clemidsogene lanparvovec) in mucopolysaccharidosis II, an ultrarare neurodegenerative disease in dire need of new therapies.
Laminins are extracellular matrix (ECM) glycoproteins that preserve the structural and functional integrity of tissues. These heterotrimeric proteins, composed of one α, β and γ chain encoded by different genes, provide mechanical support, facilitate cell adhesion and maintain tissue organization and stability.
Affinia Therapeutics Inc. has obtained IND clearance from the FDA for AFTX-201, an investigational genetic medicine for the treatment of BAG3-associated dilated cardiomyopathy (DCM). The phase I/II UPBEAT trial will begin in the first half of this year.
Zipbio has signed an exclusive license agreement with Meiragtx Holdings plc to advance an AAV gene therapy for geographic atrophy. Under the agreement, Meiragtx will receive exclusive rights to Zipbio’s first-in-class therapies targeting the complement pathway for geographic atrophy.
Another data cut from Ultragenyx Pharmaceutical Inc. regarding UX-111 (rebisufligene etisparvovec), an AAV9 gene therapy for type A Sanfilippo syndrome, continued to brighten the picture for the compound, recently taken under review again by the U.S. FDA.
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.
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