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TSHA-102 from Taysha gets rare paediatric disease and orphan drug designations for Rett syndrome expresspharma
October 16, 2020
Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.
Uni of Manchester partners with AVROBIO for Hunter syndrome gene therapy pharmatimes
October 10, 2020
The University of Manchester in the UK has partnered with US-based biotech AVROBIO on the clinical development of an investigational gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome.
Vertex, CRISPR Therapeutics’ sickle cell gene therapy wins PRIME designation pharmatimes
September 27, 2020
Vertex Pharmaceuticals and CRISPR Therapeutics have been awarded a Priority Medicines (PRIME) designation from the European Medicines Agency for their CRISPR/Cas9 gene therapy, designed to treat severe sickle cell disease (SCD).
Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by U.S. FDA prnasia
September 27, 2020
Neurophth Therapeutics, Inc., today announced that its leading candidate, NR082 (rAAV2-ND4, NFS-01 project), was granted an orphan drug designation (ODD) by the U.S. FDA for the treatment of Leber's Hereditary Optic Neuropathy associated with ND4 mutation
Vivet, Pfizer sign manufacturing pact for investigational gene therapy to treat Wilson disease expresspharma
September 24, 2020
Vivet Therapeutics and Pfizer announced that they have entered into a manufacturing agreement, under which Pfizer will provide clinical supply for a Phase 1/2 clinical trial evaluating Vivet’s proprietary, investigational gene therapy, VTX-801 ...
EU review extended for BioMarin's gene therapy Valrox pharmatimes
September 24, 2020
European regulators have requested more data to review the marketing application for BioMarin's haemophilia A gene therapy Valrox (valoctocogene roxaparvove).
Cobra Biologics and CombiGene Partner contractpharma
September 24, 2020
Sign agreement to secure GMP production of plasmids for production of CG01 gene therapy.
GenSight Biologics seeks EU approval for gene therapy pharmatimes
September 17, 2020
French biopharma company Gensight Biologics has submitted a marketing authorisation application for its lead gene therapy Lumevoq to the European Medicines Agency (EMA).
Rocket Pharmaceuticals receives FDA fast track designation for RP-L401 gene therapy for infantile malignant osteopetrosis pharmaceutical-business-review
September 01, 2020
Rocket Pharmaceuticals, a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to RP-L401..
Sunway University's Collaboration with Harvard Medical School Makes Advances in Potential Cancer Treatment using Gene Therapy prnasia
August 26, 2020
Sunway University's Professor Jeff Tan Kuan Onn of the Department of Biological Sciences and Professor Poh Chit Laa from the Centre for Virus and Vaccine Research, along with their research collaborators from Harvard Medical School's Center for Stem Cell

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