News

The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy.

The trial expects to enroll its first patient in the first half of 2023.

The decision comes after strategic uncertainty regarding a review of preliminary clinical data from GSK’s lete-cel product candidate trial.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.

Cells produced by this method were also shown to be more potent in animal models than cells made with typical processes.

The trial is being conducted at the University of California – San Diego under the leadership of Stephanie Cherqui, PhD.

As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.

The decision comes after a previous clinical hold delayed key medical milestones in the KEYNOTE-B79 trial.

The medical oncologists at Washington University School of Medicine in St. Louis discussed background to their research on associations between neurofilament light and neurotoxicity.

The DMC has recommended the FREEDOM-1 trial to continue without restrictions as the patient was treated before protocols were amended to mitigate GvHD risk.

Review top news and interview highlights from the week ending October 21, 2022.

The first 6 patients dosed in the ANTLER trial had a 50% CR rate at 6 months.

Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.

KYV-101 was developed using the construct Kyverna licensed from the NIH in January 2022.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

NKGen plans to initiate a phase 1 trial pd SNK02 in the first quarter of 2023.

With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.

The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.

The therapy is currently being evaluated in the phase 1/2 ARYA-1, ARYA-2, and ARYA-3 studies in adults and children.

The first patient has been dosed in a phase 2 study of the allogeneic CAR T-cell therapy GC007g.

In a rodent model, durable auditory brainstem responses to sound were observed after DB-OTO administration.

The director of translational research in myeloma at the Tisch Cancer Institute discussed positive findings, including a 30-month PFS in study participants.

Future data from other participants with LCA enrolled in the phase 1/2 study will be announced at a later date.

The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.

Review top news and interview highlights from the week ending October 14, 2022.

The first patient dosed with IOV-4001 has completed the safety observation period.