News

The head of the Pediatric Neurology Fellowship Program at the University of Valparaiso in Chile discussed how a lack of cost-effectiveness may hold back the potential of newborn screening in gene therapy.

A final analysis of data from CARTITUDE-1 was also presented at SOHO 2023, which revealed a PFS of 24.9 months in treated patients.

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

Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, discussed his nonclinical research on disease pathogenesis in MTLE.

Adstiladrin was approved in December 2022 and is now available for select patients under an Early Experience Program.

Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.

The chief research officer, Muscular Dystrophy Association, shared a message for Duchenne Action Month.

SynKIR-110 is based off of the KIR-CAR platform developed at the University of Pennsylvania.

The head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile discussed positive impact of newborn screening so far and work that still needs to be done.

Beam Therapeutics also expects to announce initial data from its BEACON trial in sickle cell in 2024.

The company also announced its plans for a modified study protocol that may allow a roadway to approval.

Study author Mya C. Schiess, MD, commented on trends seen in the blinded findings so far.

Zilebesiran is being evaluated as a monotherapy in the KARDIA-1 study and as a combination therapy in KARDIA-2, which will report topline data in early 2024.

ATA-100 is currently being evaluated in a multicenter phase 1/2 clinical trial (NCT05224505) in Denmark, France, and United Kingdom.

Review top news and interview highlights from the week ending September 8, 2023.

Significant improvements in ON time without troublesome dyskinesia were reported with the cell therapy after 1 year, with more apparent effects observed in the high-dose group.

In light of the IND clearance, uniQure announced its intention to carry out a phase 1/2a clinical trial, with screening of potential participants with refractory MTLE anticipated to begin in the last 3 months of 2023.

New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.

The chief development officer of Anixa Biosciences discussed the company’s trial of a CAR-T in ovarian cancer that is looking at the effect of regional administration and lymphodepletion on efficacy.

The CAR T-cell therapy, marketed as Yikaida in China, has been approved for second-line treatment for patients with refractory/relapsed large B-cell lymphoma.

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

The therapy is expected to be able to treat both the lung and liver manifestations of AATD.

The assistant professor at University College Dublin discussed challenges and different approaches to using microRNA in preclinical models of osteosarcoma.

The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.

The chief development officer of Anixa Biosciences discussed how the company is tackling 2 major challenges presented by solid tumors in its ovarian cancer clinical trial.

Stephen Gottschalk, MD, is using grant funding from ACGT to develop CAR T-cell therapy to treat children with sarcomas. He spoke about the challenges of treating sarcomas, the need for new therapies, and the importance of ACGT.

ACGT recently awarded a half-million dollar grant to brain cancer researcher Juan Fueyo, MD, who is developing an oncolytic virus therapy to treat the brain cancer glioblastoma.

September is Childhood Cancer Awareness Month, serving as a chance to assess what progress has been made in advancing cell and gene therapy research for children with cancer.

Phase 1/2 trials have been cleared in Germany and dosed first patients in the US.

Review top news and interview highlights from the week ending September 1, 2023.