Matt Hoffman

The rAAVrh8 gene therapy delivers HEXA and HEXB genes and has posted positive phase 1/2 results in infantile GM2 gangliosidosis, with enhancements in enzymatic activity and neurological outcomes.

In episode 5 of ImmunoLogic, Michael T. Lotze, MD, discusses the evolution and future of tumor-infiltrating lymphocyte therapies.

The CAR T-cell therapy showed promise in treating advanced gastric cancer by demonstrating improved survival rates and safety in pivotal trial results.

In Episode 4 of ImmunoLogic, Caroline Diorio, MD, FRCPC, FAAP, discussed her research on adverse events associated with cell therapy.

Emerging gene and cell therapies aim to slow Parkinson disease progression. Explore clinical updates on seven promising candidates in development.

In Episode 3 of ImmunoLogic, Marcela Maus, MD, PhD, discussed her research on CAR-T for treating glioblastoma.

In Episode 2 of ImmunoLogic, Bruce Levine, PhD, discussed the current the risk-benefit-ratio for CAR-T therapy.

In Episode 1 of ImmunoLogic, Stephen J. Schuster, MD, discussed the potential of treating lymphoma without chemotherapy.

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.

A long-term follow-up to the DREPAGREFFE-1 trial suggest that children with sickle cell anemia may benefit long-term on risk of cerebral injury, cognitive functions, and quality of life over standard care transfusions.

Among those who had undetectable minimal residual disease, autologous hematopoietic cell transplantation showed signs of benefit only for those who remained MRD-positive following induction therapy.

Allo-HSCT showed good 2-year survival data, with matched sibling donors showing superior outcomes to alternative donors.

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.

The Rocket Pharmaceuticals AAV9 treatment was assessed in a 3-cohort trial of 7 patients, showing good safety and encouraging efficacy signals.

Approved as Skysona, the therapy has been reported to be related to cases of hematologic malignancies, including life-threatening instances of myelodysplastic syndrome and acute myeloid leukemia.

The CAR-T therapy was approved based on the pivotal phase 1b/2 FELIX clinical trial (NCT04404660), which showed good rates of overall complete remission and median duration of remission.

Intellia’s investigational CRISPR-Cas9 gene-editing therapy NTLA-2002 reduced monthly attacks by more than 70% in both tested dose arms.

Patients with melanoma treated with ACTengine IMA203 achieved high response rates, with progression-free survival of 6 months and duration of response beyond 1 year.

The data come from the pivotal part B cohort of the phase 2/3 DEVOTE clinical trial.

Notably, the therapy previously received rare pediatric disease designation from the FDA in July 2024.

Among more than 100 patients with hemophilia A, no FVIII-specific responses showed associations with the assessed safety or efficacy parameters.

Despite the improvements, Lexeo was unable to discern a benefit on cardiovascular fitness and Peak VO2 from LX2006 with current measurements.

The medical director of clinical development at AskBio discussed setbacks in the phase 1 trial of AB-1002 gene therapy.

RG6501/OpRegen was well-tolerated, with no serious treatment-related AEs reported in follow-up data from a phase 1/2 trial.

The chief scientific officer of Caribou Biosciences discussed the company’s platform for genome editing.

Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute, pointed out that genetic forms of PD are relatively well-understood compared to other types.

The therapy, rAAV-Olig001-ASPA, has shown positive clinical benefits in several interim updates from an ongoing, first-in-human phase 1/2 clinical trial (NCT04833907).

The gene therapy is now indicated for ambulatory patients aged 4 years and older, and has been granted accelerated approval for nonambulatory patients.

In observance of World Sickle Cell Day, CGTLive brought together a variety of expert insights on navigating the rapidly expanding landscape of care for this inherited blood disorder.

Patients with large B-cell lymphoma in the TRANSFORM trial showed improvements over 3-year period compared with standard of care.