Fajar Dumadi, BSc
Regulatory Program Specialist
ISCT Head Office
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The second biannual Cell and Gene Therapy Global Regulatory Report from ISCT and Citeline will be released soon.
Following the successful release of the inaugural report in June 2023, ISCT is delighted to present the latest edition. This iteration encompasses:
- A comprehensive global overview of the regulatory landscape for cell and gene therapy, covering pipeline developments, late-stage trials (Phase III and pre-registration), and approved products.
- Highlights of significant regulatory shifts and forthcoming events, both concluded and upcoming.
- Key updates on global legislative frameworks and international standards.
Key highlights from this issue include:
- Casgevy™, the world-first gene therapy to cure Sickle Cell Disease (SCD) and Transfusion-Dependent ß-Thalassemia (TDT) utilizes the genome editing technology (CRISPR/Cas9), received its approval from EMA, MHRA, NHRA, and US FDA
- Fucaso®, the first fully human BCMA-directed CAR-T Cell Therapy for patients with relapsed or refractory Multiple Myeloma, received its approval from NMPA.
Moreover, significant strides in regulatory guidelines are evident, such as the public consultation of the US FDA Draft Guidance on Manufacturing Changes and Comparability for Human CGT Products.