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 ...

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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 ...

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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. ...

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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. ...

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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. ...

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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 ...

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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. ...

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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 ...

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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 ...

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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 ...

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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 ...

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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 ...

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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 ...

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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 ...

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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 ...

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