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.
Shares in Gensight Biologics SA dropped by as much as 41% April 7 on news that the Paris-based gene therapy firm failed to complete a second manufacturing campaign within five months, a setback that could delay a commercial launch of its lead product, Lumevoq, by at least a year.
Shares of Krystal Biotech Inc. (NASDAQ:KRYS) rocketed 121.7% higher, to $88.44 on Nov. 29, after top-line data showed its investigational gene therapy for dystrophic epidermolysis bullosa (DEB) achieved the primary endpoint of a pivotal phase III trial, healing 67% of skin wounds associated with the disease vs. placebo at six months.
Bluebird Bio Inc. has completed a rolling BLA filing of beta-thalassemia gene therapy beti-cel in the U.S., with analysts predicting that the Cambridge, Mass.-based biotech will have to shoot for a lower price tag after running into trouble with cost regulators in Europe.
DUBLIN – Bacteriophage, like other viruses, can be viewed as complex molecular machines with two essential functions: infection and replication. The first involves the efficient injection into the host cell of their genomic payload; the second involves hijacking the host’s nucleic acid replication machinery to generate the functional and structural proteins needed to give rise to progeny viruses.
