Blog
RSS FeedPosted by Michael Wonder on 15 Aug 2022
Brainstorm to seek FDA approval for ALS stem cell treatment, despite agency objections
15 August 2022 - Last year, the FDA issued a rare public statement to inform the ALS community that a ...
Posted by Michael Wonder on 15 Aug 2022
Ensuring a future for gene therapy for rare diseases
15 August 2022 - Haematopoietic stem cell gene therapy has proven to be an effective treatment for several primary immunodeficiencies, and ...
Posted by Michael Wonder on 01 Aug 2022
Gamida Cell announces FDA acceptance of biologics license application for omidubicel with priority review
1 August 2022 - PDUFA target action date is 30 January 2023. ...
Posted by Michael Wonder on 26 Jul 2022
The FDA needs to get ready to evaluate synthetic cells, the next generation of therapeutics
26 July 2022 - Expanding new medical treatments requires pushing — and sometimes breaking — the boundaries of what currently ...
Posted by Michael Wonder on 26 Jun 2022
U.S. FDA approves Bristol Myers Squibb’s CAR T cell therapy Breyanzi for relapsed or refractory large B-cell lymphoma after one prior therapy
24 June 2022 - In the pivotal Phase 3 TRANSFORM trial, single infusion of Breyanzi significantly outperformed the nearly 30 year ...
Posted by Michael Wonder on 03 Jun 2022
Gamida Cell completes rolling biologics license application submission to the FDA for omidubicel
2 June 2022 - Omidubicel has orphan drug designation and breakthrough therapy designation. ...
Posted by Michael Wonder on 01 Jun 2022
Summary of US FDA chimeric antigen receptor T-cell biologics license application approvals from a statistical perspective
25 May 2022 - The approval of tisagenlecleucel and axicabtagene ciloleucel in 2017 marked a milestone in the development of oncology ...
Posted by Michael Wonder on 28 May 2022
FDA approves Novartis Kymriah CAR-T cell therapy for adult patients with relapsed or refractory follicular lymphoma
27 May 2022 - 68% of patients receiving Kymriah in the ELARA trial experienced complete response, with an 86% overall response ...
Posted by Michael Wonder on 18 May 2022
FDA, EMA officials discuss impediments to cell and gene therapies
17 May 2022 - The US FDA's top biologics regulator said the use of a “playbook” or platform approach ...
Posted by Michael Wonder on 04 May 2022
Graphite Bio announces U.S. FDA fast track designation granted to GPH101 for the treatment of sickle cell disease
3 May 2022 - GPH101 is an investigational next-generation gene-edited therapy designed to potentially provide a one time cure for ...
Posted by Michael Wonder on 28 Apr 2022
SQZ Biotechnologies receives FDA fast track designation for its lead cell therapy candidate for the treatment of HPV16 positive tumours
27 April 2022 - Designation Creates Potential to Bring Important New Therapy to Patients Earlier. ...
Posted by Michael Wonder on 27 Apr 2022
Editas Medicine receives FDA rare paediatric disease designation for EDIT-301 for the treatment of beta thalassaemia
26 April 2022 - Editas Medicine today announced that the U.S. FDA granted rare paediatric disease designation to EDIT-301, an investigational, ...
Posted by Michael Wonder on 26 Apr 2022
VBL Therapeutics receives FDA fast track designation for ofra-vec for the treatment of platinum-resistant ovarian cancer
26 April 2022 - OVAL Phase 3 top-line progression-free survival primary outcome data for ofra-vec expected in 2H 2022; with ...
Posted by Michael Wonder on 25 Apr 2022
FDA grants regenerative medicine advanced therapy designation to Autolus’ CAR T cell therapy, obe-cel, for the treatment of adult B-ALL
25 April 2022 - Autolus Therapeutics today announced that the U.S. FDA has granted regenerative medicine advanced therapy designation to its ...
Posted by Michael Wonder on 20 Apr 2022
FDA grants regenerative medicine advanced therapy designation to AlloVir’s posoleucel for prevention of multiple life-threatening infections from six viruses in allogeneic haematopoietic cell transplant patients
20 April 2022 - Posoleucel’s third regenerative medicine advanced therapy designation marks an unprecedented regulatory distinction among cell and gene therapies. ...