Noah Stansfield

Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, discussed potential upcoming innovations in the field.

The director of cell therapy and transplant at Penn Medicine discussed progress made and experienced gained for the field of cell therapy for autoimmune disease in 2024.

The CRL was related inspection of a third-party manufacturer rather than any efficacy or safety concerns regarding the product itself.

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed the importance of referring patients with r/r LBCL who are transplant ineligible for CAR-T treatment.

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

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed the choice between treating patients with liso-cel or bispecific T-cell engagers.

In addition to the new designation, ViGeneron also announced that the data safety monitoring board for the trial has given the green light for dose escalation.

The vice president research and chair of internal medicine at the University of Erlangen – Nuremberg discussed potential upcoming innovations in the field.

The multicenter trial is open to patients with forms of r/r B-NHL including DLBCL, FL, MCL, and MZL.

Among 9 patients treated in the study who were evaluable at 2 years posttreatment, all 9 showed improved or preserved visual function in comparison to their untreated fellow eyes.

The clinical director of lymphoma services at the University of Colorado discussed the importance of referring patients with r/r LBCL who are transplant ineligible for CAR-T treatment.

The company’s phase 1/2 LIGHTHOUSE clinical trial for ATSN-201 has moved onto its Part B.

The clinical director of lymphoma services at the University of Colorado discussed the choice between treating patients with liso-cel or bispecific T-cell engagers.

According to EsoBiotec, this is the first time a patient has been treated with an in vivo BCMA-directed CAR-T therapy in a clinical trial.

The data comes from 3 patients treated at the trial’s low dose.

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

Restem-L previously received orphan drug designation from the FDA for PM/DM in December 2024.

Ultragenyx is seeking accelerated approval for the BLA based on the use of CSF heparan sulfate data from the phase 1/2/3 Transpher A clinical trial.

Arbor noted it has designed redePHine, a phase 1/2 clinical trial (NCT identifier pending) that will evaluate ABO-101 in adults and children with PH1.

Cell therapy, particularly CAR-T, is expanding into autoimmune diseases like lupus and multiple sclerosis. While promising, safety, efficacy, and broad applicability remain uncertain.

The company requested priority review for the BLA.

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed 5-year follow-up data from the TRANSCEND-NHL-001 clinical trial.

The trial is evaluating AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.

Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia, discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

The product will be marketed under the name Ryoncil, and is indicated for patients aged 2 months and older.

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

The agency agreed that data from the ongoing phase 1/2 studies compared to external control natural history data will be sufficient to support a BLA.

Hosted by Joseph Fraietta, PhD, ImmunoLogic will be tailored for an audience fluent in the language of medicine and biotechnology, offering data-driven insights while maintaining accessibility.

The trial for the exon-51 skipping therapy is already active in the United Kingdom.