Rocket Pharmaceuticals Inc. has received IND clearance from the FDA for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM). RP-A601 offers the potential for a one-time, curative alternative to medical therapy, implantable cardioverter defibrillators and ablations.
A proof of concept of ex vivo genetic modification of cells from patients and their transplantation in mice has demonstrated, for the first time, the therapeutic possibilities of prime editing in sickle cell disease (SCD).
Atsena Therapeutics Inc.’s IND application for ATSN-201 has been cleared by the FDA enabling the company to initiate a phase I/II trial in patients with X-linked retinoschisis (XLRS) caused by pathogenic or likely pathogenic mutations in RS1.
Ocugen Inc.’s OCU-410ST (AAV5-hRORA) has been awarded orphan drug designation by the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19) and cone-rod dystrophy 3 (CORD3) diseases.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
Base editing (BE), a technique that modifies a single nucleotide in living cells, has been successfully tested to resolve the CD3δ mutation in severe combined immunodeficiencies (SCIDs) and produce functional T cells. For now, scientists at the University of California, Los Angeles (UCLA), completed the study on patient stem cells and artificial thymic organoids, shortening the way for future clinical trials.
A modification of the CRISPR technique has made it possible to restore vision in mouse models with retinitis pigmentosa (RP). Scientists at the Institute of Visual Neuroscience and Stem Cell Engineering of Wuhan University of Science and Technology developed a new gene-editing tool called PE(SpRY) to edit in vivo a mutation of enzyme phosphodiesterase 6B (PDE6β) and return its function.
Osteoarthritis and its associated cartilage pathology affects 30 million people in the U.S., but no disease-modifying treatments have yet reached the clinic. A recent multicenter trial evaluating the safety and efficacy of a truncated, recombinant human fibroblast growth factor-18 (FGF18) protein analogue (rhFGF18) demonstrated a dose-dependent improvement in cartilage thickness relative to a placebo.
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