Blog
RSS FeedPosted by Michael Wonder on 28 Sep 2022
CRISPR Therapeutics announces FDA regenerative medicine advanced therapy designation granted to CTX130 for the treatment of cutaneous T-cell lymphomas
28 September 2022 - CRISPR Therapeutics today announced that the US FDA granted regenerative medicine advanced therapy designation to CTX130, ...
Posted by Michael Wonder on 31 Aug 2022
US FDA grants fast track designation for Longeveron’s Lomecel-B product for treatment of hypoplastic left heart syndrome in infants
31 August 2022 - New designation may expedite FDA review and potential approval to address this life-threatening heart condition affecting ...
Posted by Michael Wonder on 25 Aug 2022
Iovance Biotherapeutics initiates biologics license application submission for lifileucel in advanced melanoma
25 August 2022 - First TIL therapy BLA submission initiated with U.S. FDA. ...
Posted by Michael Wonder on 17 Aug 2022
bluebird bio announces U.S. commercial infrastructure to enable patient access to Zynteglo, the first and only FDA approved gene therapy for people with beta thalassaemia who require regular red blood cell transfusions
17 August 2022 - Innovative outcomes-based contract offering includes single upfront payment and up to 80% risk-sharing. ...
Posted by Michael Wonder on 17 Aug 2022
FDA approves first cell based gene therapy to treat adult and paediatric patients with beta thalassaemia who require regular blood transfusions
17 August 2022 - Today, the US FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of ...
Posted 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 ...