In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss, 10 out of 11 have shown improvements after being treated with a gene therapy. “What is really remarkable about this type of therapeutic approach is that the first people who are going to see the impact are not actually the physicians – it’s the families,” Jonathon Whitton, vice president and Regeneron’s auditory global program head, told BioWorld.
Gene therapy specialist Meiragtx Holdings plc is heading for its first marketing approval following the successful treatment of 11 children with Leber amaurosis, a severe form of congenital retinal dystrophy that rendered them blind at birth. The 11 children, aged between 1 and 4 years old, all gained visual acuity following a single delivery of a correct version of the AIPL1 (aryl-hydrocarbon interacting protein-like1) gene, which codes for a photoreceptor protein in the cones and rods.
The longstanding ambition of developing an inhaled gene therapy for cystic fibrosis has taken a step forward, with the start of a phase I/II trial of a product using a novel pseudotyped viral vector that it is hoped will circumvent problems encountered in previous studies with other vectors.
EG 427 SAS has closed a €27 million (US$28.3 million) series B round, which will fund it to completion of the first clinical trial of the lead gene therapy program, opening the way for its herpes simplex viral-vectored products to be developed in a range of chronic neuro-urology disorders.
Researchers from Tikkunlev Therapeutics Inc. and the University of Utah have presented preclinical data on TLT-101, a gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector encoding cardiac bridging integrator 1 (cBIN1) designed for the treatment of heart failure.
Solid Biosciences Inc. is preparing for a sit-down with the U.S. FDA this year to discuss the firm’s results with the next-generation gene therapy SGT-003 for Duchenne muscular dystrophy (DMD).
Multiple sulfatase deficiency is an autosomal recessive lysosomal storage disorder caused by homozygous or compound heterozygous loss-of-function mutations in the SUMF1 gene, which encodes formylglycine generating enzyme (FGE), which catalyzes the post-translational modification of all known 17 sulfatases.
Voyager Therapeutics Inc. has announced its decision to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). Emerging preclinical data indicate the siRNA payload component of VY-9323 does not meet its standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window.
4D Molecular Therapeutics Inc.’s gene therapy, 4D-150, in wet age-related macular degeneration (wet AMD) produced positive phase IIb data as the company preps two phase III studies set to begin this year.
Arbor Biotechnologies Inc.’s ABO-101 has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of primary hyperoxaluria type 1 (PH1).
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