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).
Entos Pharmaceuticals Inc. has been awarded a $4 million grant by California Institute for Regenerative Medicine (CIRM) to support the completion of IND-enabling activities with ENTLEP-001, a durable genetic medicine for the treatment of congenital generalized lipodystrophy.
Navega Therapeutics Inc. has received a $4 million Translational Science grant from the California Institute for Regenerative Medicine (CIRM) to support its work addressing neuropathic pain. The grant will fund the final preclinical development studies of NT-Z001 leading to an IND submission.
California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the development of a hematopoietic stem cell (HSC) gene therapy to treat Angelman syndrome, including preparation of a pre-IND package submission to the FDA.
Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular and Clinical Ophthalmology Basel and collaborators proposed a novel dual AAV vector system to deliver a split-intein adenine base editor for precise correction of the most common Stargardt mutation, c.5882G>A (p.Gly1961Glu).
Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.
A 6.5-month-old boy with the rare inherited urea cycle disorder ornithine transcarbamylase (OTC) deficiency has responded positively in a targeted in vivo gene editing trial, in which a correct copy of a defective gene was inserted at a precise locus in the genome.
Yoltech Therapeutics Co. Ltd. has advanced YOLT-204 into the clinic for the treatment of transfusion-dependent β-thalassemia (TDT). If successful, YOLT-204 may provide an off-the-shelf curative treatment for TDT patients without conditioning chemotherapy and hematopoietic stem cell transplantation.
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