For International Batten Awareness Day, held annually on June 9, CGTLive reached out to several experts to inquire about the potential impact of gene therapy on the Batten disease treatment landscape.
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Updated data were presented at the ISCT 2023 Meeting.
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The FDA’s Advisory Committee meeting is scheduled for September 27, 2023, and the PDUFA date for the company’s BLA review is set for December 8, 2023.
The associate professor at Medical College of Wisconsin discussed the rationale for the CARTITUDE-4 study and its latest updates.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The study has enrolled 17 patients as of May 2023 and enrollment is expected to complete by the end of 2024.
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The REVEAL trial is being carried out in Canada under a CTA that was cleared by Health Canada in March 2022.
New overall survival analysis data on Yescarta from ZUMA-7 were presented at the ASCO 2023 meeting.
The associate professor at University College London also discussed unmet needs that remain in r/r B-ALL and another presentation of interest at ASCO’s 2023 conference.
The professor of medicine and pediatrics at Washington University in St. Louis discussed evaluating afami-cel in the SPEARHEAD-1 trial.
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Among 19 patients with r/r B-ALL included in the analysis, all 19 achieved a CR or CRi.
Safety findings were also improved when compared with the previous CARTITUDE-1 study, suggesting the therapy may be better tolerated in earlier lines of therapy.
The director of the Lymphoma Clinical Research Program at University of Texas MD Anderson Cancer Center discussed the implications of overall survival data that he presented at ASCO’s 2023 conference.
The study began enrollment in January 2023.
Additionally, promising efficacy signals were reported—namely response rates and disease control rates—in the phase 1 clinical trial (NCT04429087) data that were presented at the 2023 ASCO Annual Meeting.
The professor of medicine and pediatrics at Washington University in St. Louis discussed updated survival data from SPEARHEAD-1.
The associate professor in haemato-oncology at University College London discussed Autolus Therapeutics’ CAR-T, which utilizes a novel fast off-rate CD19 binding domain.
Among 94 patients who were treated with obe-cel in the trial, 76% achieved a CR or CRi.
The autologous CAR T-cell therapy A2B530 is being investigated for solid tumors in the multicenter, first-in-human, phase 1/2 EVEREST-1 study.
Among 70 patients who were able to be evaluated for efficacy at 28 days or more, 91% achieved a complete response or a complete response with incomplete hematological recovery.
Follow-up data were presented from the discontinued phase 3 OVAL study.
There were no DLTs observed among the treated patients.
Maria Pia Morelli, MD, PhD, assistant professor at MD Anderson Cancer Center, discussed the first-in-human study of Sleeping Beauty TCR-T cells.
Among 49 patients who were included in the trial’s primary efficacy analysis set and received dose-level 2, the CR/CRi rate was 18.4%.
Review top news and interview highlights from the week ending June 2, 2023.
The chief scientific officer at Omega Therapeutics discussed positive preclinical data presented at ASCO 2023.
Genprex also announced preliminary data from the phase 1 portion of the trial evaluating Reqorsa, which is being presented at ASCO’s 2023 meeting.
In more positive news, the company’s phase 2 trial for treating DMD will enroll patients in the second half of 2023.
This is the second IND clearance for CABA-201, which previously received clearance for evaluation in systemic lupus erythematosus, and now for active idiopathic inflammatory myopathy—also known as myositis.
The scientist at Senti Biosciences discussed developing small molecule-regulated gene circuits.
