Cell & Gene Therapy
Uncover the transformative potential of cell and gene therapies. Delve into groundbreaking research, clinical advancements, and regulatory developments in this cutting-edge field. Stay informed about the latest breakthroughs and their impact on patient care and disease treatment.
First cell treatment for type I diabetes is approved by the FDA
Source – FDA On June 29, 2023, CellTrans achieved a significant milestone by securing the first-ever FDA approval for a ...
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 Pfizer’s 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 ...
Kite Successfully Transfers Marketing Authorization for Yescarta CAR T-cell Therapy in Japan
Source – Gilead Sciences According to Kite Pharma on June 22, 2023, the marketing authorization for Yescarta, a chimeric antigen ...
FDA Grants Priority Review for Bluebird Bio’s Sickle Cell Treatment
Source – bluebird bio On June 21, 2023, the US Food and Drug Administration (FDA) accepted the Biologics Licence Application ...
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 ...
FDA Sets December Deadline for Vertex and CRISPR’s Sickle Cell Therapy
The US Food and Drug Administration (FDA) has initiated a priority review of exagamglogene autotemcel (exa-cel), a therapy developed by ...
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. ...
