The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating adeno-associated virus (AAV) gene replacement therapy AT-845 in adults with late-onset Pompe disease.
After long years of painstaking work, the commercialization of cell and gene therapies picked up pace in 2022, with multiple approvals. More progress is expected in 2023, with several firsts in the offing and products for larger patient populations reaching the market.
It has been shown that site-specific phosphorylation of tau at threonine 205 (T205) by the kinase p38γ disengages tau from toxic pathways, thus acting as a neuroprotective function in Alzheimer’s disease. Researchers in Australia have designed a novel AAV-based gene therapy approach targeting p38γ, DBA/2.p38γCA, in murine models of epilepsy in which tau is known to play a neurotoxic effect.
Sonothera Inc. reeled in $60.75 million in a series A financing led by Arch Venture Partners. The funds are earmarked for the continued development of Sonothera’s ultrasound-guided, nonviral, gene therapy platform and treatments.
Coave Therapeutics SA has entered into a collaboration with the Institute of Neurodegenerative Diseases (IMN), a joint research unit associating the University of Bordeaux and the French National Centre of Scientific Research (CNRS), to develop gene therapy programs targeting protein degradation in neurodegenerative disorders.
Akouos Inc. has received IND clearance from the FDA to initiate a first-in-human phase I/II pediatric trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss.
Australia’s Therapeutic Goods Administration (TGA) should better align its definitions and classifications for gene, cell and tissue frameworks with international regulators, stakeholders said.
Australia’s Therapeutic Goods Administration (TGA) should better align its definitions and classifications for gene, cell and tissue frameworks with international regulators, stakeholders said.
Although there has been huge progress in treatment of cystic fibrosis over the last decade, with Vertex Pharmaceuticals Inc. becoming the first to address the underlying cause of the disease with its Kalydeco (ivacaftor), approved in 2012, there are still many patients who aren’t eligible for treatment.
The first gene therapy to treat severe hemophilia A was among the drugs recommended for European approval by regulators from the EMA’s CHMP at its monthly meeting. Manufactured by Biomarin Pharmaceutical Inc., Roctavian (valoctocogene roxaparvovec) was recommended for conditional marketing authorization in the EU for severe hemophilia A in adults who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5.
