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RSS FeedPosted 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 16 Nov 2023
Vertex and CRISPR Therapeutics announce authorisation of the first CRISPR/Cas9 gene edited therapy, Casgevy (exagamglogene autotemcel), by the United Kingdom MHRA for the treatment of sickle cell disease and transfusion dependent beta thalassaemia
16 November 2023 - First regulatory authorisation of a CRISPR-based gene editing therapy in the world. ...
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 09 Nov 2023
Paddy Gower has issues: blind, solo mum desperate for PHARMAC to fund life-changing medicine so she can see her kids
8 November 2023 - We sat in a field of wild daisies. Bright white and yellow against a backdrop of ...
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 06 Nov 2023
Expanded reimbursement for Spinraza improves patient access
2 November 2023 - Biogen Korea is strengthening its position in the local spinal muscular atrophy market with expanded reimbursement ...
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 ...
Posted by Michael Wonder on 30 Oct 2023
ICER publishes final evidence report on gene therapy for metachromatic leukodystrophy
30 October 2023 - Independent appraisal committee voted that across all patient sub-populations, arsa-cel demonstrated a net health benefit when ...
Posted by Michael Wonder on 27 Oct 2023
Health Canada authorises CSL's Hemgenix (etranacogene dezaparvovec) as first gene therapy for haemophilia B
26 October 2023 - CSL's Hemgenix is a one time, single dose treatment for adults with haemophilia B who require ...
Posted by Michael Wonder on 23 Oct 2023
Life changing gene therapy for babies with spinal muscular atrophy
23 October 2023 - The Australian Government is giving more families access to life changing gene therapy for spinal muscular atrophy ...