Blog
RSS FeedPosted by Michael Wonder on 23 Dec 2023
Sarepta Therapeutics submits efficacy supplement to expand the Elevidys label to include Duchenne muscular dystrophy patients without restriction to age or ambulatory status
22 December 2023 - Sarepta has also submitted the EMBARK post-marketing requirement to the FDA seeking conversion of the Elevidys accelerated ...
Posted by Michael Wonder on 22 Dec 2023
Wainua (eplontersen) granted first ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis
21 December 2023 - US FDA approval based on NEURO-TTRansform Phase 3 results showing Wainua demonstrated consistent and sustained benefit improving ...
Posted by Michael Wonder on 20 Dec 2023
Ocugen announces OCU400 receives regenerative medicine advanced therapy designation for treatment of retinitis pigmentosa associated with RHO mutations
19 December 2023 - Ocugen today announced that the FDA has granted regenerative medicine advanced therapy designation to Ocugen’s investigational product ...
Posted by Michael Wonder on 18 Dec 2023
Lexeo Therapeutics granted FDA fast track designation and orphan drug designation for LX2020, an AAV based gene therapy candidate for PKP2 arrhythmogenic cardiomyopathy
18 December 2023 - Lexeo Therapeutics today announced the US FDA has granted fast track designation and orphan drug designation to ...
Posted by Michael Wonder on 12 Dec 2023
Candel Therapeutics receives FDA fast track designation for CAN-2409 in pancreatic cancer
12 December 2023 - Candel Therapeutics today announced that the US FDA granted fast track designation for its lead investigational adenovirus ...
Posted by Michael Wonder on 12 Dec 2023
FDA creates new advisory committee for evaluation of genetic metabolic disease treatments
12 December 2023 - Today, the US FDA announced it is creating a new advisory committee related to potential treatments for ...
Posted by Michael Wonder on 08 Dec 2023
Statement from President Joe Biden on FDA approval of gene therapies to treat sickle cell disease
8 December 2023 - As we’ve seen throughout our history, when the US Government invests in innovation, we can achieve breakthroughs ...
Posted by Michael Wonder on 08 Dec 2023
FDA approves first gene therapies to treat patients with sickle cell disease
8 December 2023 - Today, the US FDA approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies ...
Posted by Michael Wonder on 07 Dec 2023
Solid Biosciences receives FDA fast track designation for Duchenne muscular dystrophy gene therapy SGT-003
7 December 2023 - Next generation gene transfer therapy to treat Duchenne receives FDA fast track Designation. ...
Posted by Michael Wonder on 05 Dec 2023
CG Oncology receives both FDA fast track and breakthrough therapy designation for cretostimogene grenadenorepvec in high risk BCG unresponsive non-muscle invasive bladder cancer
5 December 2023 - Recent Phase 3 monotherapy first results demonstrated complete response rate of 75.7% at any time. ...
Posted by Michael Wonder on 28 Nov 2023
Abeona Therapeutics announces FDA accepts and grants priority review for Pz-cel biologics license application
27 November 2023 - PDUFA target action date is 25 May 2024. ...
Posted by Michael Wonder on 20 Nov 2023
The FDA is at a crossroads on cell and gene therapies
20 November 2023 - Cell and gene therapies are the next frontier in medicine and promise long-sought hope for people ...
Posted by Michael Wonder on 14 Nov 2023
Atsena Therapeutics receives FDA regenerative medicine advanced therapy designation for ATSN-101 gene therapy for GUCY2D associated Leber congenital amaurosis
14 November 2023 - ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious ...
Posted by Michael Wonder on 06 Nov 2023
AviadoBio announces FDA IND clearance and fast track designation for AVB-101 for the treatment of frontotemporal dementia with progranulin mutations
6 November 2023 - AVB-101 is an investigational gene therapy delivered as a one time infusion directly to the brain, enabling ...
Posted by Michael Wonder on 31 Oct 2023
US FDA awards both rare paediatric disease and orphan drug designations to HG204, a CRISPR RNA editing therapy, for the treatment of MECP2 duplication syndrome
31 October 2023 - HG204 is the world first CRISPR RNA-editing therapy for the treatment of MECP2 duplication syndrome granted ...