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.
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.
Affinia Therapeutics Inc. has nominated AFTX-201 as a development candidate for the treatment of BAG3 dilated cardiomyopathy. The gene therapy, using Affinia’s cardiotropic capsid, is given as a one-time intravenous injection.
About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.