相关临床试验
36
10 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
1919
进行中（未招募）
9
25.0%
已完成
18
50.0%
尚未招募
1
2.8%
招募中
6
16.7%
暂无批准数据
- REGENXBIO Inc. appointed Greg Ciongoli, Founder and Managing Partner of Adiumentum Capital Management, to its Board of Directors effective August 25, 2026. - Mr. Ciongoli brings over 16 years of biotechnology investing and capital allocation experience from The Baupost Group to support REGENXBIO's transition into a global commercial organization. - Concurrent with the appointment, independent directors Jerry Karabelas, Ph.D., and Jean Bennett, M.D., Ph.D., retired from the Board after years of distinguished service. - The appointment strengthens the Board as REGENXBIO advances a late-stage pipeline of potential first- and best-in-class AAV gene therapies.
- Capricor Therapeutics will release its second quarter 2026 financial results after market close on Thursday, August 13, 2026. - Management will host a conference call and webcast at 4:30 p.m. ET the same day to discuss results and provide a recent corporate update. - The company's lead candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage development for Duchenne muscular dystrophy (DMD). - Capricor is also advancing its StealthX exosome platform for targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics.
- Nippon Shinyaku has exercised its exclusive U.S. commercialization option for Tadekinig alfa, triggering a $30 million payment to AB2 Bio following positive FDA interactions. - Tadekinig alfa targets Primary Monogenic IL-18-Driven Hyperinflammatory Syndrome caused by NLRC4 and XIAP mutations, an ultra-rare pediatric disease with no approved therapies. - AB2 Bio is eligible to receive up to $600 million in total milestones and royalties, while retaining worldwide rights for all other indications and ex-U.S. rights for the lead indication. - The recombinant IL-18 binding protein has completed its Phase 3 program and holds Breakthrough Therapy, Rare Pediatric Disease, and Orphan Drug designations from the FDA.
- Nippon Shinyaku has exercised its option rights to obtain exclusive U.S. commercialization rights for Tadekinig alfa from AB2 BIO Ltd., building on a January 2025 option agreement. - Tadekinig alfa targets monogenic IL-18 driven Hyperinflammatory Syndrome in patients with NLRC4 and XIAP mutations, two rare hereditary autoinflammatory diseases with no currently approved therapies. - The recombinant human IL-18 binding protein has received Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations in the United States. - Following U.S. regulatory approval, NS Pharma, a wholly owned subsidiary of Nippon Shinyaku, is expected to lead commercialization efforts for this underserved patient population.
- Elixirgen Therapeutics and Nippon Shinyaku have entered into an option agreement granting Nippon Shinyaku potential exclusive worldwide commercialization rights to EXG-7001 for Duchenne muscular dystrophy. - EXG-7001 is a locally administered, full-length dystrophin mRNA therapeutic currently in preclinical development, designed to deliver the complete dystrophin protein regardless of a patient's genetic mutation. - Nippon Shinyaku will fund developmental costs, while Elixirgen receives an upfront payment and is eligible for additional development and sales-based milestone payments if the option is exercised. - Current DMD therapies focus on delivering or restoring an incomplete dystrophin protein, leaving a significant unmet need for a therapy capable of delivering the full-length protein.
- NS Pharma and NCNP presented 4.5-year clinical trial data for brogidirsen, an antisense oligonucleotide therapy for Duchenne muscular dystrophy patients with exon 44 skipping mutations. - Six participants maintained motor function scores on North Star Ambulatory Assessment and Performance of Upper Limb assessments throughout the extended treatment period. - The therapy demonstrated an acceptable safety profile with no serious adverse events related to long-term administration and no treatment discontinuations over 4.5 years. - Results support brogidirsen's potential to modify DMD disease progression, with a second global Phase II study currently underway to further evaluate efficacy.
- Atsena Therapeutics has completed dosing in Part B of the LIGHTHOUSE trial for ATSN-201, a gene therapy treating X-linked retinoschisis, with no serious adverse events reported. - The company plans to initiate enrollment of the pivotal Part C cohort in Q1 2026, which will evaluate 56 adult and pediatric patients across treatment and control groups. - ATSN-201 represents the first XLRS gene therapy to demonstrate efficacy in clinical trials, with patients showing improvements in retinal structure and visual function. - The therapy targets approximately 30,000 males in the U.S. and EU who suffer from XLRS, a condition with no currently approved treatments.
- The Duchenne Muscular Dystrophy market reached approximately $2.15 billion in 2023 and is expected to grow significantly due to increased drug uptake and anticipated gene therapy launches. - Over 75 companies are actively developing pipeline therapies for DMD, with recent FDA designations including Atossa Therapeutics' (Z)-Endoxifen receiving Rare Pediatric Disease designation. - Capricor Therapeutics announced positive Phase 3 HOPE-3 trial results for Deramiocel, while the FDA accepted their BLA for review with Priority Review designation. - Current approved treatments include EMFLAZA, VYONDYS 53, EXONDYS 51, AMONDYS 45, VILTEPSO, and gene therapy ELEVIDYS in the US, with AGAMREE launched in Germany in 2024.
- Capricor Therapeutics published a peer-reviewed study in Biomedicines describing a novel in-vitro potency assay that characterizes the anti-fibrotic mechanism of action of Deramiocel for Duchenne muscular dystrophy treatment. - The study demonstrated that cardiosphere-derived cells suppress collagen I and III gene expression in primary human fibroblasts through secreted exosomes and soluble factors, with consistent results across over 100 manufacturing lots. - The validated assay enhances quality control and product consistency for Deramiocel as it advances through late-stage development, with Phase 3 HOPE-3 trial topline data expected in mid-fourth quarter 2025. - Deramiocel has received multiple regulatory designations including Orphan Drug status from FDA and EMA, and Regenerative Medicine Advanced Therapy designation, supporting its potential for treating this rare neuromuscular disorder.
- NS Pharma and NCNP presented 3.5-year clinical trial data for brogidirsen, an antisense oligonucleotide therapy for Duchenne muscular dystrophy patients amenable to exon 44 skipping. - The study demonstrated high exon 44 skipping efficiency and sustained dystrophin expression levels in muscle biopsies at both week 25/26 and week 99/100. - Ambulant participants maintained motor function over the long-term treatment period with no serious adverse events or treatment discontinuations reported. - The findings support brogidirsen's potential to modify DMD disease progression, with a global Phase II study currently underway.
