相关临床试验
7
4 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
N/A
进行中（未招募）
4
57.1%
招募中
2
28.6%
撤回
1
14.3%
暂无批准数据
- Atamyo Therapeutics announced encouraging safety and efficacy results for ATA-200 gene therapy in the first four patients with LGMD-R5, a severe childhood muscular dystrophy that leads to loss of walking ability before adulthood. - The Phase 1b/2 trial showed over 90% of muscle fibers expressing the therapeutic protein in the first two patients at six months, with significant reductions in muscle damage biomarkers and clinical improvements in functional tests at nine months. - No serious adverse events were observed in the four treated patients, demonstrating the safety profile of the single intravenous AAV-based gene therapy targeting gamma-sarcoglycan deficiency. - The results represent rare biological data in neuromuscular diseases at such an early trial stage, offering potential hope for approximately 2,000 patients with LGMD-2C/R5 in Europe and the US who currently have no curative treatment options.
- The FDA has cleared Atamyo Therapeutics' IND application for ATA-200, a gene therapy for Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5). - ATA-200 delivers a functional copy of the SGCG gene and has shown promise in preclinical studies, correcting symptoms and disease biomarkers in mice. - The Phase 1b dose-escalation trial (NCT05973630) will now expand to the US, supported by funding from The Dion Foundation, with plans to open the first US center before year-end. - ATA-200 has also been granted orphan drug designation by the FDA, highlighting the unmet need for treatments for this rare pediatric disease.
- The FDA has approved Atamyo Therapeutics' Investigational New Drug application for ATA-200, a gene therapy for limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). - ATA-200 is the first potential treatment for LGMD2C/R5 to enter clinical development in the U.S., offering hope for children with this debilitating condition. - The Phase 1b trial will enroll approximately six children aged 6-13 across sites in the U.S., France and Italy, evaluating the safety and tolerability of ATA-200. - ATA-200 delivers a healthy copy of the _SGCG_ gene to muscle cells, aiming to restore gamma-sarcoglycan protein production and improve muscle function in affected individuals.
• The FDA has approved obecabtagene autoleucel (obe-cel), a CD19-directed CAR-T therapy, for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-cell ALL). • Approval was based on the Phase 1b/2 FELIX trial, which demonstrated overall complete remission rates above 60% and durable remissions exceeding 12 months. • Clinical holds on CARsgen's BCMA-directed CAR-T therapy zevorcabtagene autoleucel and Claudin18.2-directed CAR-T satricabtagene autoleucel have been removed by the FDA.
- Atamyo Therapeutics completed recruitment for the dose escalation phase of the Phase 1b trial of ATA-100 for LGMD2I/R9. - The company submitted an IND application to the FDA for ATA-200, a gene therapy for LGMD2C/R5, expanding its clinical trial to the U.S. - Initial results from the ATA-100 trial show promising functional improvements and a marked decline in creatine kinase levels in treated patients. - The FDA granted rare pediatric disease designation to ATA-200, highlighting the unmet need for treatments for these rare muscular dystrophies.
