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RFK Jr looks to alter rare disease regulation by fast-tracking drug approvals

Pharmaceutical Technology

The approval process for cell and gene therapy candidates is tricky, however, given their high-cost and oft-debated benefit-risk ratio. RFK Jr did not question their importance, and acknowledged panellists’ calls for faster regulatory processes. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Harbour BioMed partners Otsuka on new T-cell therapy

Pharmaceutical Technology

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. In 2024, Otsuka announced the acquisition of US-based Jnana Therapeutics in a deal worth up to $1.1bn. Editorial content is independently produced and follows the highest standards of journalistic integrity.

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Health Care Riders

Putting Patients First Blog

Since it is likely this appropriations package will be one of the last meaningful pieces of legislation before the November elections, other policy priorities may be added to that final legislation as it gets closer to passage. We reiterated these priorities to start the year in January 2024. The House passed H.R.

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SpliceBio secures $135m for gene therapy development

Pharmaceutical Technology

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. In 2023, SpliceBio signed a licensing agreement with Spark Therapeutics, allowing the latter to use its protein splicing platform and develop a gene therapy for an undisclosed inherited renal disease.

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DoH and Children’s National Hospital link for cell and gene therapy

Pharmaceutical Technology

The Department of Health – Abu Dhabi (DoH) in the United Arab Emirates (UAE) and US Children’s National Hospital (known as the Children’s National) have signed a memorandum of understanding (MoU) to advance paediatric cell and gene therapy. How will RFK Jr’s American dream for vaccines play out?

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NSW government invests $13.39m in life-changing therapies

Pharmaceutical Technology

June 20, 2025 Share Copy Link Share on X Share on Linkedin Share on Facebook The treatments can be accessed through facilities such as Royal Prince Alfred Hospital, Westmead Hospital and the Sydney Children’s Hospitals Network. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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JCR Pharma and Alexion partner on JUST-AAV gene therapy platform

Pharmaceutical Technology

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. How will RFK Jr’s American dream for vaccines play out? JCR has also developed a proprietary blood-brain barrier-penetrating technology, J-Brain Cargo, to deliver biotherapeutics into the central nervous system.