Victoria Johnson

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

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 head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris Cité discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.

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.

Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.

The 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 Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.

Sana Biotechnology’s SC291 chimeric antigen receptor T-cell therapy is being developed to treat relapsed/refractory systemic lupus erythematosus, including extrarenal lupus and lupus nephritis.

Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, discussed findings from 2 early studies of CD19 CAR T-cell therapy.

The vice president research and chair of internal medicine at the University of Erlangen – Nuremberg discussed findings from 2 early studies of CD19 CAR T-cell therapy.

As cell therapy investigations mature in the field of AD, some programs show promising signs of efficacy.

Atsena announced initial positive data from the LIGHTHOUSE study of ATSN-201 in May 2024.

A patient with MG is also the first evaluated to reach 1 year of follow-up and has a continued durable immunomodulator-free response.

The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.

TRX103 is also being evaluated for the prevention of graft versus host disease (GvHD) in patients undergoing HLA-mismatched HSCT.

The chief medical officer at Creyon Bio discussed future applications for the company’s AI-guided discovery platform.

NGN-401 is being evaluated in an early phase clinical trial which has been selected for the FDA’s START Pilot Program.

Elderly patients were at higher risk of infections and subsequent malignant neoplasms.

Participants are being dosed with OCU400 in the ongoing phase 3 liMeliGhT trial.

The cofounder and chief science officer of Longeveron discussed working to show potential benefits in a follow-up phase 3 trial.

The trial evaluating the safety and tolerability of IDP-023 will be led by Stanford and UCSF.

A recent study also recommended a shorter, more flexible monitoring period post-CAR–T therapy.

The chief medical officer at Creyon Bio discussed findings from a treated patient with a TNP02 missense mutation.

BriaCell initiated the pivotal phase 3 BRIA-ABC trial in October 2023.

At the selected phase 2 dose, 76% of patients were injection free, with maintained visual acuity and fluid control.

BioNTech is evaluating BNT111 in a phase 2 trial, primary analysis data from which will be presented in a future medical conference.

The company has filed a protocol amendment with the EMA and anticipates resuming recruitment imminently.

OBX-115 recently demonstrated a 50% ORR at doses over 30 × 109 cells at 29.5 weeks after infusion.

There were no new cases of CRS past 2 weeks after infusion and non-relapse mortality was driven by infection in follow-up.