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With promising data, ProQR Therapeutics' RNA-based eye drug heads to phase 3 fiercebiotech
September 06, 2018
ProQR’s treatment, QR-110, is designed to repair the genetic defect underlying LCA10 in the RNA, which could potentially stop disease progression or even reverse some of its effects.
Gene therapy receives Champalimaud Vision Award europeanpharmaceuticalreview
September 05, 2018
The Champalimaud Vision Award has been awarded to research teams for the development of a gene therapy…
uniQure starts dosing in pre-pivotal hemophilia B gene therapy trial fiercebiotech
August 27, 2018
Dutch biotech uniQure has treated the first of around three patients due to enroll in its phase 2b dose-confirmation trial of hemophilia B gene therapy AMT-061.
Mustang Bio licenses gene therapy for bubble boy disease pharmaceutical-technology
August 16, 2018
US-based biopharmaceutical firm Mustang Bio has entered an exclusive worldwide licence agreement with St. Jude Children’s Research Hospital for a new gene therapy to treat X-linked severe combined immunodeficiency (X-SCID or bubble boy disease).
Orchard raises $150m to advance gene therapies pharmatimes
August 15, 2018
UK-American biotech Orchard Therapeutics has raised $150 million to fuel development of its gene therapy pipeline.
Orchard Therapeutics raises $150 M to improve Gene Therapy Pipeline biospectrumasia
August 14, 2018
Proceeds to advance three late-stage programs towards registration, prepare for commercialization and progress the company’s gene therapy platform
Mustang Bio picks up St. Jude’s ‘bubble boy’ gene therapy fiercebiotech
August 14, 2018
Mustang Bio has licensed a gene therapy from St. Jude Children’s Research Hospital for X-linked severe combined immunodeficiency (X-SCID), also called “bubble boy” syndrome.
Fresh from GSK deal, Orchard raises $150M for multifront late-phase gene therapy push fiercebiotech
August 14, 2018
Orchard Therapeutics has raised $150 million (€132 million) to advance three gene therapies toward approval. The series C round continues a transformative year for Orchard defined by the acquisition of GlaxoSmithKline’s portfolio of approved and experimen
UC Berkeley backs oral gene therapy company targeting the gut in $1.5M seed round fiercebiotech
August 13, 2018
A UC Berkeley-backed preclinical company, DNAlite Therapeutics, has gathered $1.5 million in seed money to pursue oral gene therapies that can slip through the mucus barrier that typically blocks access to the gastrointestinal tract.
Sarepta builds in gene therapy with $30M Lacerta deal fiercebiotech
August 10, 2018
Sarepta isn’t allowing an ongoing issue with its lead gene therapy candidate to dampen its enthusiasm for the field. It just announced a tie-up with Lacerta will add another three programs to the eight already on its books and extends its focus beyond mus

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