Gene Therapy
Krystal’s Vyjuvek Gene Therapy Gains FDA Approval, Spurs Sales Projections
Krystal Biotech’s CEO, Krish Krishnan, was quick to highlight in the company’s inaugural conference call to present its quarterly results ...
Catalent Braces for Potential Revenue Setback Following Sarepta’s Gene Therapy Trial Disappointment
Catalent, having overcome the challenges posed by the pandemic, now faces another potential setback in the form of what analysts ...
Taysha halts gene therapy for rare nerve disease after FDA and Astellas feedback
Astellas has declined the opportunity to exercise an option for an asset from Taysha Gene Therapies. Taysha, facing study design ...
Rocket Pharma gets FDA green light for gene therapy trial, stock surges
Rocket Pharmaceuticals has achieved alignment with the FDA regarding the design of a pivotal phase 2 clinical trial for a ...
BioMarin R&D chief reveals two new pipeline candidates and strategy to sustain innovation
BioMarin, the California-based biotech giant, is embarking on a mission to create its most robust pipeline in history, with a ...
Ferring launches bladder cancer gene therapy Adstiladrin in US through early experience program
Ferring Pharmaceuticals achieved a significant milestone by administering its groundbreaking gene therapy, Adstiladrin, to its first commercial patient. This innovative ...
Novartis drops Gyroscopeās gene therapy for eye disease after disappointing trial results
Novartis is in the process of divesting its eye disease unit, a move that includes discontinuing a geographic atrophy (GA) ...
Discover the rare yet aggressive uterine sarcoma, its symptoms, causes, and treatments.
A cancer called uterine sarcoma develops in the uterus’s (the womb’s) muscle or other tissues. It is different from endometrial ...
Thermo Fisher Cuts Over 200 Jobs at Clinical-Stage Cell and Gene Therapy Facility in Florida
In the midst of ongoing workforce reductions impacting the biotech sector, even prominent players like Bristol Myers Squibb, Emergent BioSolutions, ...
Gene Therapy Vector Lawsuit: Henrietta Lacks’ Estate Targets Ultragenyx Over Exploitation
Just under a month after reaching a settlement with Thermo Fisher over the utilization of Henrietta Lacks’ cells, her family ...
Astellas Buys $50M Stake in Poseida, Boosting Gene Therapy Pipeline
Source – Poseida Therapeutics Japanese pharmaceutical company Astellas is making a strategic move by investing $50 million to acquire an ...
Despite CSL’s hemophilia B gene therapy, NICE is not persuaded
The UK’s National Institute for Health and Care Excellence (NICE) has expressed hesitation in recommending approval for CSL Behring and ...
Sensorion Seeks Approval for Clinical Trial of OTOF-GT Gene Therapy Candidate in the UK
Source – Sensorion On July 10, Sensorion filed its first regulatory application to start human testing of OTOF-GT, a gene ...
BioMarin’s Roctavian: FDA Approves Hemophilia A Gene Therapy with Positive Reception from Physician
Source – BioMarin BioMarin’s Roctavian, a gene therapy for severe hemophilia A, has received FDA approval after initial rejection in ...
FDA Accepts Pfizerās Application for Hemophilia B Gene Therapy Fidanacogene Elaparvovec
Source – Pfizer On 28 June 2023, Pfizer announced that the US Food and Drug Administration (FDA) and the European ...
FDA’s Peter Marks Overrides Reviewers’ Rejection to Approve Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy
Despite the FDA review teams initially leaning against approving Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy, a memo reveals that ...
uniQure craters on the gene therapy data from Huntington Disease
Source –uniQure On June 21, 2023 uniQure announced promising interim data, including up to 24 months of follow-up, from 26 ...
Sarepta Therapeutics Makes History with FDA Approval of Elevidys, the First Gene Therapy for Duchenne Muscular Dystrophy
Source – Sarepta Therapeutics On June 22, 2023, Sarepta Therapeutics made an announcement regarding the accelerated approval granted by the ...
Sarepta’s Breakthrough DMD Gene Treatment, Elevidys, Triumphs FDA Hurdles at $3.2M
The speedy approval of Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy has been achieved after several delays and a close ...
High Cost of New Cancer Drugs and Lack of Awareness Impede Adoption in the US
A new survey conducted by healthcare research firm Sermo revealed that oncologists in the United States perceive pricing as the ...
Analysts predict that BioMarin’s hemophilia A gene therapy may succeed in Europe
According to a report published by SVB Securities analysts Joseph Schwartz and Joori Park, Ph.D., Roctavian may have a “significant ...
Unlocking the Future of Healthcare: The Rising Power of Gene Therapy
Gene therapy, a promising field at the intersection of genetics and medicine, is revolutionizing the way we approach disease treatment. ...
