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The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed 3-year follow-up data from the HOPE B trial of the approved gene therapy, branded as Hemgenix.

BCMA-directed CAR T-cell therapy anitocabtagene autoleucel demonstrated a 76% complete remission rate and 89% negative minimal residual disease in patients with relapsed/refractory multiple myeloma.

The chief medical officer of Orca Bio discussed data the company presented on its Orca-T and Orca-Q programs at ASH 2023.

The CSL Behring/uniQure gene therapy produced significant reductions in annualized bleeding rates and the number of total bleeds, with a consistent safety profile and no new adverse events.

The associate professor at Peter MacCallum Cancer Centre discussed case study research from the center and possible contributions to the case.

No significant differences were observed between patients with and without HCBV in the reductions seen in ABR for all bleeds during the 7 to 36 months posttreatment timeframe.

Among patients with chronic lymphocytic leukemia and small lymphocytic lymphoma, the Bristol Myers Squibb CAR T cell product showed consistent efficacy in patients with a lack of success with BTK inhibitor and venetoclax treatment.

The professor of hematology at University of Lille and chair of LYSA shared updated data from the TRANSCEND-FL trial.

The bone marrow transplant physician at St. Jude Children’s Research Hospital also discussed the recent approval of exa-cel.

A CRISPR/Cas9-edited allogeneic stem cell transplantation effectively reduced hematopoietic toxicity associated with maintenance gemtuzumab ozogamicin for high-risk acute myeloid leukemia (AML).

Data from 3 early phase clinical trials assessing BRL-101 in 10 patients were presented at the ASH 2023 meeting.

CD19-directed CAR-T-cell therapy resulted in sustained remission for over a year and no return of autoimmunity for patients with systemic lupus erythematosus, idiopathic inflammatory myositis, and systemic sclerosis.

The bone marrow transplant physician at St. Jude Children’s Research Hospital discussed an analysis of the sickle cell disease gene therapy he’s presenting on at ASH’s 2023 meeting.

Long-term data on lovo-cel gene therapy for sickle cell disease (SCD) reveals sustained efficacy at 60 months.

Bluebird bio’s gene therapy, marketed as Zynteglo, showed successful rates of transfusion independence up to 9 years with a reasonable safety profile in data presented at ASH 2023.

There were no treatment-related serious adverse events reported.

The FDA’s Nicole Verdun, MD; and Peter Marks, MD, PhD, offered insight on the recent approvals of exa-cel and lovo-cel, answering questions about the safety of the gene therapies and the continued collection of data on their use.

Review top news and interview highlights from the week ending December 8, 2023.

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185).

The FDA’s decision was based on data from the CLIMB-121 and CLIMB-131 clinical trials, making it the first CRISPR-based gene therapy to be approved in the US.

The president and head of R&D at Taysha Gene Therapies discussed the early clinical evaluation of TSHA-102.

Benetic anticipates an announcement of interim results sometime around the midpoint of 2024.

The associate professor of dermatology at Stanford University discussed his experiences investigating and helping to develop Vyjuvek, approved for treating DEB.

Stempeutics mesenchymal stem cell therapy is approved in India for treating critical limb ischemia due to multiple causes.

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

The cell therapy yielded a median overall survival of around 17 months compared with a historical OS of less than 12 months.

The director of cell therapy and transplant at Penn Medicine discussed the seminar he gave at the inaugural Cell Therapy for Autoimmune Disease Summit.

ORR was 40% in the 10-mg group and 32% in the 100-mg group.