
Cell Therapy New Drug Developer

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Focus on the Next Generation of CAR-T Therapy: Lyell Immunopharma Acquires ImmPACT Bio
Lyell Immunopharma today announced that it has entered into a definitive agreement to acquire ImmPACT Bio.ImmPACT Bio's lead investigational therapy, IMPT-314, is a CD19/CD20-targeted chimeric antigen receptor (CAR) T-cell therapy, and Lyell Immunopharma will continue to advance the development of this product in hematologic malignancies, including large B-cell lymphoma.IMPT-314 employs a dual-targeting design aimed at surpassing the efficacy of approved CD19-targeted CAR-T cell therapies and enhancing the persistence of CAR-T cell therapy by enriching naïve and central memory T cells during the manufacturing process.The acquisition of ImmPACT is expected to significantly enhance Lyell’s clinical-stage next-generation CAR-T cell therapy pipeline and complement its proprietary technology portfolio to generate more durable and functional T cells, providing longer-lasting efficacy for patients with solid tumors and hematologic malignancies.

Clinical trial data released earlier this year showed,Among 13 patients with relapsed/refractory (R/R) aggressive non-Hodgkin lymphoma treated with IMPT-314, 12 achieved complete or partial responses (92% objective response rate), with 10 patients achieving confirmed complete responses (CR), resulting in a complete response rate of 77%.At a median follow-up time of 32 months, the median progression-free survival was 50.1 months, and the median overall survival has not yet been reached.
BridgeBio Pharma Gene Therapy Significantly Improves Patient Outcomes
Recently, BridgeBio Pharma announced the latest data from the phase 1/2 clinical trial, CANaspire, evaluating the investigational gene therapy BBP-812 for the treatment of Canavan disease. The trial results showed that BBP-812 significantly improved biomarker levels in patients. Compared to natural history data, treated patients also demonstrated progressive and sustained improvements in motor function. The press release noted that if approved,BBP-812 may becomeCanavan DiseaseThe First Approved Therapy for Patients.

CANaspire is a Phase 1/2 open-label study designed to evaluate BBP-812 in patients withCanavan Diseasein pediatric patients for safety, tolerability, and pharmacodynamic activity. The trial results showed,Following administration of BBP-812, levels of N-acetylaspartate (NAA) in the urine and brain of participants rapidly and persistently decreased. At 12 months post low-dose treatment, NAA levels had decreased by an average of 64%, and at 3 months post high-dose treatment, NAA levels had decreased by an average of 73%.The earliest participants to receive treatment maintained low levels of urinary NAA for nearly three years.NAA in cerebrospinal fluid (CSF) decreased by an average of 70% after 12 months of low-dose treatment. In T2-weighted magnetic resonance imaging (MRI), improved myelination was observed in most participants treated with BBP-812.Compared with the natural history, patients treated with BBP-812 showed gradual improvement in motor function (assessed by GMFM-88 scale) and reached motor function milestones (evaluated by HINE-2 scale).
Canavan DiseaseIs a kind of aspartic acid enzyme encoded byASPAUltra-rare, disabling or life-threatening diseases caused by genetic mutations.Most children are unable to crawl, walk, sit, or speak, and face the threat of death during infancy. The lack of aspartoacylase activity leads to the accumulation of NAA, which eventually causes toxicity to myelin. Currently, there is no approved therapy for this disease.BBP-812 uses adeno-associated virus 9 vector toASPAFunctional copies of the gene are delivered throughout the body and into the brain, potentially offering a cure for the disease.It has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation, Rare Pediatric Disease designation, and Fast Track designation by the U.S. FDA.
Treating Fibrotic Diseases: Newcomer Completes $89 Million Financing
Agomab Therapeutics Announces $89 Million Series D Financing to Advance Clinical Development of Lead Candidate AGMB-129




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