The EMA has been sent back to the drawing board to re-evaluate PTC Therapeutics Inc.’s Duchenne muscular dystrophy therapy Translarna (ataluren), after failing to get the usual rubber stamp following its recommendation in January that the drug’s conditional approval be withdrawn.
Researchers from Purespring Therapeutics Ltd. and affiliated organizations have presented preclinical data for the adeno-associated vector (AAV) gene therapy PS-001 for the treatment of glomerular disease.
Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for gene therapy DTX-401 as the first potential medical treatment for glycogen storage disease type 1a (GSD1a) after the phase III study hit its primary endpoint and two key secondary endpoints.
Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common mutation being the E342K mutation (Z allele) that introduces an amino acid change from glutamic acid (E) coding for M-AAT to a lysine (K) coding for Z-AAT.
Avirmax Biopharma Inc. has received IND approval from the FDA to initiate a phase I/IIa trial for its gene therapy treatment targeting wet age-related macular degeneration (AMD), including polypoidal choroidal vasculopathy (PCV).
Kate Therapeutics Inc. recently presented data on a novel muscle- and heart-targeted, liver de-targeted development candidate for the treatment of Duchenne muscular dystrophy (DMD) – KT-809.
A boy participating in the phase II Daylight study of Duchenne muscular dystrophy (DMD) “has passed away suddenly,” according to Pfizer Inc. The participant had received fordadistrogene movaparvovec, a mini-dystrophin gene therapy, in early 2023. The fatal serious adverse event was reported May 3 as a cardiac arrest, Pfizer told BioWorld. Pfizer, together with the independent external data monitoring committee, is reviewing the data to understand the potential cause, the company added.
Philadelphia-based Latus Bio Inc., co-founded by serial biotech entrepreneurs P. Peter Ghoroghchian and Beverly Davidson, launched on May 2 with two lead adeno-associated virus (AAV)-based gene therapy candidates and $54 million in a series A financing.
Ending a late 2021 deal that was potentially worth billions, Moderna Inc. and Metagenomi Inc. are going their separate ways. The two had been collaborating on gene-editing R&D to develop therapies for treating serious genetic diseases. Moderna said it agreed with Metagenomi to end the deal as “Moderna continues to strategically prioritize its research and development investments.”
Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
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