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Boehringer forms new alliance to develop gene therapy pharmaceutical-technology
August 08, 2018
Boehringer Ingelheim has entered a global partnership with the UK Cystic Fibrosis Gene Therapy Consortium (GTC), Imperial Innovations and Oxford BioMedica (OXB) to develop a new long-term gene therapy for cystic fibrosis (CF).
Akouos raises $50M to take hearing loss gene therapy into humans fiercebiotech
August 08, 2018
Akouos has raised $50 million to take a gene therapy treatment for hearing loss into the clinic. The Boston-based biotech unveiled the series A round alongside the appointment of a Spark Therapeutics co-founder and other organizational changes.
Quality blip halts Sarepta muscular dystrophy gene therapy trial fiercebiotech
July 30, 2018
A phase 1/2a trial Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has been placed on clinical hold by the FDA after rogue DNA was found in a test sample.
PTC inks $200M Agilis takeover to snag late-phase gene therapy fiercebiotech
July 26, 2018
PTC Therapeutics is paying $200 million upfront to acquire Agilis Biotherapeutics. The deal gives PTC ownership of a gene therapy treatment for rare neurotransmitter disease AADC deficiency that is set to go before the FDA next year.
Amryt in-licenses novel gene therapy platform from UCD pharmatimes
July 23, 2018
UK biotech Amryt has secured itself access to a novel gene therapy platform that could give rise to a new treatment option for patients with Epidermolysis bullosa (EB), a group of rare inherited skin disorders that cause the skin to become very fragile.
Novartis snaps up AveXis in $8.7bn deal pharmatimes
July 17, 2018
Novartis is buying US gene therapy group AveXis in a deal valued at $8.7 billion.
Controlling CRISPR and gene therapy with an amino acid switch fiercebiotech
July 09, 2018
Gene editing and gene therapy have a range of potential applications, from editing out disease-causing mutations to treating spinal cord injuries.
Plasticell leads gene therapy manufacturing consortium biospectrumasia
June 27, 2018
UK’s innovation agency provides funding to develop advanced technologies for the manufacturing of autologous ex vivo gene therapies.
Axovant pivots with new gene therapy, Spark co-founder as CTO fiercebiotech
June 11, 2018
After a disastrous year in which its Alzheimer’s attempts blew up and the once biotech star David Hung jumped ship, Axovant is continuing its change-up with a new focus and a new executive.
Gene therapy startup MeiraGTx files for $86M IPO fiercebiotech
May 16, 2018
MeiraGTx has filed for an $86 million IPO to advance a handful of gene therapies into and through clinical trials.

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