Noah Stansfield

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

The 2-part, multicenter BCT-006-US trial will seek to enroll patients who are in the earlier stages of ALS.

AURN001 consists of allogeneic human corneal endothelial cells referred to as “neltependocel” and Y-27632, a small molecule drug.

Atul Malhotra, MD, PhD, the head of the early neurodevelopment clinic at Monash Children's Hospital, discussed his lab’s research in the field of neonatal cell therapy.

Caribou stated that it anticipates initiating its planned phase 1 GALLOP clinical trial for CB-010 in patients with LN and ERL by the end of 2024.

Haydar Frangoul, MD, discussed advantages and disadvantages of haploidentical bone marrow transplant and the 2 new gene therapies for SCD.

Eque-cel was previously evaluated in antibody-mediated idiopathic inflammatory disorders of the nervous system in an investigator-initiated trial.

Nirav Shah, MD, an associate professor of medicine at Medical College of Wisconsin, discussed LV20.19, a bispecific CD19/CD20-targeted CAR-T being evaluated for chronic lymphocytic leukemia and Richter’s transformation.

The FDA’s decision was based on data from the phase 3 CARTITUDE-4 clinical trial (NCT04181827).

Uttam Rao, MD, MBA, a transplant physician at St. David's South Austin Medical Center of Sarah Cannon, discussed research comparing patient outcomes on different conditioning regimens for CAR-T.

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

Atul Malhotra, MD, PhD, the head of the early neurodevelopment clinic at Monash Children's Hospital, discussed the importance of preclinical guidelines in the context of neonatal cell therapy.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed how there is still much room for growth for RNA therapeutics despite progress so far.

In observance of Parkinson's Awareness Month, held annually in April, CGTLive® took a closer look at REGENERATE-PD, a new trial for PD gene therapy AB-1005.

Vivien Sheehan, MD, PhD, an associate professor of pediatrics at Emory University, discussed how patients should be able to choose to try the recently FDA-approved cell-based therapies for SCD when they are ready.

In light of the CTA clearance, Atamyo plans to carry out a multicenter, open-label phase 1b clinical trial (NCT05973630).

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed her research on siRNA technology.

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

Notably, the combination therapy, referred to as SYNCAR-001 + STK-009, will be administered in the trial without the use of lymphodepletion.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the importance of further research into the root cause of MS and other autoimmune diseases.

The company noted that it plans to submit the BLA in the second quarter of 2024.

John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his view of the main priorities for research in this area.

Bemdaneprocel remained well-tolerated and no major safety issues had occurred in the treated patients.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed considerations for evaluating CAR-T in patients most likely to obtain meaningful benefit.

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

John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his team’s early findings on CAR-T and fertility from CIBMTR centers.

Arsa-cel, approved under the name Lenmeldy, is set to be priced at $4.25 million.

At the 2-month follow-up PET/CT scan, the patient showed a 6.6% reduction in tumor size.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the potential impact of CAR-T therapy on the lives of patients with autoimmune disease.

Sandra P. Reyna, MD, the chief scientific advisor and head of global medical engagement for SMA at Novartis, discussed data from the phase 3b SMART clinical trial (NCT04851873) that were presented at MDA's 2024 conference.