Personalized therapies for diabetes, while showing promise in international studies, is met with healthy skepticism from ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine This voice experience is generated by AI. Learn more. This ...

Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and clinicians in Lund have now presented a new model for cooperation that will ...

Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.

Center for Drug Evaluation, National Medical Products Administration, Beijing, China. Clinical Trial Center for Drugs and Medical Devices, Beijing Tsinghua Changgung Hospital, School of Clinical ...

In this webinar, Stefan Radtke will discuss the development of new high-throughput single-cell readouts for precise and comprehensive tracking of gene editing outcomes after autologous hematopoietic ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Consider the case of patient WB, a 79-year-old male with recurrent diffuse large B cell lymphoma. He was referred for ...

Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...