相关临床试验
5
1 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
2016
已完成
1
20.0%
尚未招募
1
20.0%
招募中
2
40.0%
暂停
1
20.0%
暂无批准数据
- Alltrna has received approval to begin a Phase 1 trial of AP003 in healthy volunteers in Australia, which the company describes as the first tRNA therapy to enter human clinical trials. - AP003 is a lipid nanoparticle-encapsulated tRNA delivered by IV infusion that is designed to read through premature stop signals caused by nonsense mutations. - The company targets the Arg-to-stop mutation, the most common nonsense mutation, within a universe of roughly 19 conserved stop signals that recur across many genetic diseases. - Alltrna plans an adult phenylketonuria study to show protein restoration and biomarker reduction before a basket trial in multiple inborn errors of metabolism.
- Entrada Therapeutics selected ENTR-801 as its first clinical candidate for ocular diseases, targeting Usher syndrome type 2A through exon 13 skipping to restore usherin protein production. - The company expects multiple data readouts in 2026 from its Duchenne muscular dystrophy franchise, including ELEVATE-44-201 data in Q2 2026 and ELEVATE-45-201 data in mid-2026. - Entrada maintains a robust cash runway into Q3 2027 and plans to nominate a second clinical candidate in ocular diseases during 2026.
- Taiho Pharmaceutical's pizuglanstat (TAS-205) failed to demonstrate significant improvement in time to rise from floor compared to placebo in the Phase III REACH-DMD study. - The randomized, double-blind trial enrolled 82 male DMD patients aged 5 years and older across 26 Japanese sites over three years. - This setback adds to recent DMD therapeutic failures, including Sarepta/Roche's Elevidys and Pfizer's withdrawn gene therapy program. - Pizuglanstat works by inhibiting hematopoietic prostaglandin D synthase to reduce muscle necrosis and inflammatory response in DMD patients.
• Phase I/II Deliver trial demonstrates sustained functional improvements in DMD patients treated with DYNE-251, showing mean absolute dystrophin expression of 8.72% above baseline at six months with the 20mg/kg dose. • The therapy, designed for patients amenable to exon 51 skipping, showed improvements across multiple functional endpoints including mobility assessments, positioning Dyne for potential accelerated FDA approval submission in early 2026. • If approved, DYNE-251 could generate significant market impact, with GlobalData projecting revenue growth from $5 million in 2025 to $129 million by 2030 in the competitive DMD treatment landscape.
- Entrada Therapeutics has received MHRA authorization to commence ELEVATE-44-201, a Phase I/II trial evaluating ENTR-601-44 for Duchenne muscular dystrophy patients with exon 44 skipping mutations. - The global trial consists of two parts: a multiple ascending dose study with 24 subjects receiving 6-18mg/kg doses, followed by an efficacy and safety assessment phase with quality-of-life measures. - ENTR-601-44, an Endosomal Escape Vehicle-conjugated therapy, aims to restore functional dystrophin protein production, with trial initiation planned for Q2 2024.
