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Gene Therapy (AAV) · news · pharmadog

← back to Gene Therapy (AAV)

News · Gene Therapy (AAV)

216 stories

17hUltragenyx, after setbacks, nabs first gene therapy approvalbiopharma dive

18hSTAT+: How a Trump loyalist got the FDA nominationstat

19hSTAT+: FDA approves Ultragenyx gene therapystat

19hSTAT+: Pharmalittle: We’re reading about a Regeneron rare disease drug, an Ultragenyx gene therapy, and morestat

20hUltragenyx bags first gene therapy approval as previously rejected Sanfilippo asset awaits decisionbiospace

21hFDA approves Ultragenyx's gene therapy Genglycos for glycogen storage disorderendpoints

1dRecon: FDA approves Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation challengesraps

1dFDA finalizes Q&A guidance on developing cell and gene therapiesraps

1dFDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Iafda press

1dKolon TissueGene cuts 37 US staffers after Phase 3 failbiospace

1d11 companies hiring in New Jerseybiospace

2dSTAT+: In wake of boy’s death, gene editing experts dissect Huidagene’s decisionsstat

2dKolon TissueGene shrinks workforce after pivotal osteoarthritis trial missfiercebiotech

2dLeo Pharma buys rare skin disease drug in $435M dealbiopharma dive

2dLEO adds to dermatology portfolio with $435M deal for late-stage rare disease drugbiospace

3dAssessing shared failures with AI is key to cell and gene therapy’s evolutionbiospace

4dLeo ‘really strengthening’ pipeline with $435M deal for Tanabe rare skin disease drug awaiting FDA nodfiercebiotech

6dAfter 2 undisclosed gene therapy deaths, can China’s alternative regulatory path still be trusted?fiercebiotech

6dCapricor soars on plan to amend DMD filing after adcomm setbackfiercebiotech

7dPTC takes a chance on Sangamo’s Fabry disease gene therapybiopharma dive

7dBankrupt Sangamo offloads assets to Lilly, PTC in $161M salebiospace

8dPTC’s $211M bid wins Sangamo auction, teeing up ‘special opportunity’ to enter Fabry marketfiercebiotech

8dSTAT+: Definium Therapeutics says its LSD therapy eased anxiety in late-stage trialstat

8dSTAT+: Biotech company co-founded by top autoimmune researcher lays out plans with antibody therapiesstat

9dSTAT+: Neurocrine Biosciences rare disease drug possibly tied to safety issues, experts saystat

9dSTAT+: Trump is counting on the health care industry to remake vaccines. It’s pushing back insteadstat

9dEpicrispr, Infinimmune snag combined $165M in VC cashbiospace

9dSTAT+: Skylark Bio kicks off new gene therapy trial for deafnessstat

10dIRBs have a problem. Can North Star’s nonprofit model save it?stat

10dScribe’s Benjamin Oakes on a rare IPO and bringing CRISPR to the massesbiopharma dive

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