News

The chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed the panel he participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.

At the 24-week analysis, patients who received the high dose achieved an 89% decrease in annualized antiVEGF injection rates.

Review top news and interview highlights from the week ending February 9, 2024.

The gene-edited cell therapy also showed a safety profile consistent with that previously seen in patients with early-onset MLD.

Data from defunct company Lysogene’s discontinued trial of LYS-GM101 were presented at WORLDSymposium.

Among the 5 patients treated in the study, sustained supraphysiological expression of the disease-targeted gene, SGSH, was rapidly achieved in the leukocytes and the plasma.

The chief scientific officer at Omega Therapeutics discussed how the MYCHELANGEO-1 trial may validate the company’s OEC platform.

Hematological recovery was achieved and maintained in all 4 patients in the study.

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

Orchard Therapeutics has also randomized the first patient with Hurler Syndrome in the phase 3, registrational HURCULES trial of OTL-203 gene therapy.

ETX101 consists of a transgene encoding a GABAergic regulatory element and an engineered transcription factor that is delivered via a nonreplicating, recombinant AAV9 vector.

CDR-Life also recently presented positive preclinical data at the November 2023 SITC Annual Meeting.

The associate professor of dermatology at Stanford University discussed the potential impact of Vyjuvek’s approval in the derm field.

The new Cell and Gene Therapy Access Model will be led by the Centers for Medicare & Medicaid Services’ Innovation Center.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed MDA Care Centers and his excitement for the organization’s the Upcoming Clinical & Scientific Conference.

AK-OTOF delivers transgenes encoding OTOF, the disease-targeted gene, to the inner hair cells of the cochlea via a single unilateral intracochlear administration.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive™ team.

For World Cancer Day, held annually on February 4, take a look at the stories that have stood out in oncology cell therapy so far this year.

Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed future plans for the expanded applications of the allogeneic regenerative neural cell therapy.

The Prescription Drug User Fee Act (PDUFA) target action date has been set for August 4, 2024.

Review top news and interview highlights from the week ending February 2, 2024.

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed work the National Alliance of Sickle Cell Centers is conducting.

The ongoing, multistage phase 1/2/3 Cyprus2+ study (NCT04884815) expects to read out initial safety and efficacy data in the first half of 2024.

Vor Bio recently also announced data from the phase 1/2 trial of trem-cel.

The Associate Professor of Medicine at Harvard Medical School discussed CAR T’s journey to treating solid tumors.

Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed the ongoing first-in-human clinical trial evaluating the cell therapy for drug-resistant mesial temporal lobe epilepsy.

The associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.

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

Kyverna has also announced data on its proprietary CAR-T manufacturing process, Ingenui-T.

Efficacy and safety results from the RESKUE clinical trial will be presented at at the WORLDSymposium 2024 on February 9.