Latest Conference Coverage

The treatment seeks to improve immune reconstitution and graft-versus-leukemia immune response without increasing the risk of GvHD in children and young adults with hematologic malignancies.

Preliminary findings from the phase 1 clinical trial demonstrate good cell persistence and expansion in vivo.

The real-world data support the recently published results from a phase 3 study, which showed most patients achieved transfusion independence.

The greatest improvements were seen in the Pain, Fatigue, and Physical Functioning domains.

In 37 evaluable patients, the objective response rate in the study was 89% (95% CI, 75%-97%) with complete responses in 78%.

In this late-breaking poster presented at the Tandem Meetings, investigators recommend that patients with primary or secondary central nervous system lymphoma be included in future clinical trials for CAR T-cell therapy.

Armed with an average of 14.3 months of follow-up data, investigators share updates from a cohort in the phase 2 study evaluating cilta-cel in lenalidomide-refractory patients with progressive multiple myeloma after 1-3 prior lines of therapy at the 2022 Tandem Meetings.

Shephard Mpofu, MD, part of the Novartis Gene Therapies leadership, shared his thoughts on the latest phase 3 data on onasemnogene abeparvovec (Zolgensma) from the SPR1NT trial.

Peter J. McAllister, MD, FAAN, presented promising data from the phase 2 STEMTRA trial at AAN 2022.

The phase 1, investigator-initiated trial continues to recruit participants with B-ALL.

The novel regimen from BioNTech demonstrated encouraging results in patients with testicular or ovarian cancer.

AFM13 is currently also being evaluated on its own in lymphoma in a phase 2 registrational study.

The first in-human phase 1 clinical trial is being conducted in Japan.

Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.

Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.

New data presented at MDA 2022 showcased positive motor and bulbar function data.

The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD

The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed potential effects of cilta-cel's approval.

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.

The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.

The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.

The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.

The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.

The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.

The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.

The trial design was informed by both patient and physician perspectives.