相关临床试验
44
14 进行中
药物批准
10
批准总数
监管机构
2
监管机构数
成立时间
N/A
进行中（未招募）
7
15.9%
已完成
24
54.5%
尚未招募
7
15.9%
招募中
4
9.1%
撤回
2
4.5%
- Vertex reported positive Phase 2b AMPLIFIED results for inaxaplin in two APOL1-mediated kidney disease populations not enrolled in its pivotal AMPLITUDE trial. - In the modest proteinuria cohort, inaxaplin 45 mg once daily reduced urine albumin-to-creatinine ratio by 42.7% at Week 13 versus baseline. - The type 2 diabetes cohort showed a smaller 17.3% UACR reduction, with the confidence interval crossing zero, and UPCR fell 25.4%. - Vertex has completed full enrollment in the Phase 2/3 AMPLITUDE study, with interim analysis data expected in early 2027.
- Vertex Pharmaceuticals withdrew EU applications to extend Kaftrio and Kalydeco combination treatment to cystic fibrosis patients aged one year and older. - The EMA published notice on September 18 that Vertex Pharmaceuticals (Ireland) Limited pulled the Kalydeco (ivacaftor) extension, with the withdrawal dated September 1, 2026. - A parallel Kaftrio (ivacaftor/tezacaftor/elexacaftor) granule application for the same age extension was also withdrawn after EMA preliminary feedback requested more information.
- Under CEO Reshma Kewalramani, Vertex launched Casgevy, the first FDA-approved CRISPR gene-editing drug, and Journavx, a new class of non-opioid painkillers. - Vertex posted $12 billion in profit in 2025 with a market capitalization exceeding $100 billion, expanding beyond cystic fibrosis into neurological, hematological, and renal diseases. - Bahija Jallal rescued Immunocore from near-collapse, raised $130 million, and secured FDA approval for Kimmtrak, the world's first therapy for inoperable or metastatic uveal melanoma. - GSK's US president Maya Martinez-Davis oversaw five new drug launches in 2025, while Teal Health's Kara Egan brought the first FDA-approved at-home cervical cancer screening kit to market.
- ReCode Therapeutics received new funding from the Cystic Fibrosis Foundation and entered a research collaboration with an undisclosed gene editing company to develop CFTR-targeted genetic medicines. - The collaboration combines ReCode's SORT LNP delivery platform with the partner's gene editing technology, aiming to advance candidates toward clinical development and commercialization. - Heather Clark, a nearly 30-year rare disease drug development veteran who helped develop five FDA-approved CF therapies at Vertex, assumed the CEO role effective July 1, 2026. - ReCode's inhaled mRNA therapy RCT2100, which received FDA Fast Track designation in February 2026, has a fully enrolled Phase 2a study with data expected in Q4.
- Mana.bio has appointed Dr. Laura Sepp-Lorenzino, former CSO of Intellia Therapeutics, to its Scientific Advisory Board to guide the company's AI-driven LNP discovery platform. - Dr. Sepp-Lorenzino brings decades of leadership experience from Intellia, Vertex Pharmaceuticals, Alnylam, and Merck, spanning CRISPR, RNAi, and nucleic acid therapies. - Mana.bio's proprietary AI/ML platform leverages the world's largest curated LNP dataset to predict safety, tropism, and stability, with NHP-validated delivery solutions for extrahepatic targets. - The appointment comes at what CEO Yogev Debbi describes as a pivotal stage of growth as the company scales its discovery engine and expands its therapeutic pipeline.
- Cryoport's total Q2 2026 revenue reached $49.0 million, an 8% year-over-year increase driven by 15% growth in Life Sciences Services and 25% growth in BioStorage/BioServices. - The company achieved positive adjusted EBITDA from continuing operations of $0.4 million, marking an important milestone on its pathway to sustainable profitability. - Cryoport now supports a record 779 global clinical trials and 22 commercially approved cell and gene therapies as of June 30, 2026. - Four customers filed BLA/MAA applications during the quarter, and the company anticipates up to 11 additional filings and five potential new therapy approvals for the remainder of 2026.
- Latigo Biotherapeutics filed for an IPO on Nasdaq under ticker "LTGO" to fund its pipeline of non-opioid Nav1.8 sodium channel inhibitors for acute and chronic pain. - Lead candidate LTG-001 met the primary endpoint in a 343-patient abdominoplasty trial, showing statistically significant pain reduction versus placebo and numerically outperforming hydrocodone/acetaminophen. - The company believes this study could serve as one of two pivotal trials required for FDA approval, with Phase III bunionectomy trials expected by end of year. - Latigo has raised approximately $322 million since inception and aims to compete alongside Vertex's approved Journavx and Eli Lilly's acquired SiteOne program in the emerging Nav1.8 market.
- Two new industry reports from ResearchAndMarkets.com provide comprehensive analysis of over 1,859 clinical-stage partnering agreements and 1,567 royalty-disclosing licensing transactions in the pharmaceutical and biotechnology sectors. - The clinical-stage report covers deal structures, upfront payments, milestone triggers, and royalty rates across Phase I, II, and III agreements, with contract documents where available. - The royalty rates report examines financial terms across 1,567 licensing transactions since 2015, offering benchmarking data on royalty clauses, license fees, and payment structures. - Both resources are designed to help biopharma dealmakers benchmark deal structures, optimize negotiation strategies, and assess partner flexibility in clinical-stage and royalty-based collaborations.
- Casgevy (exagamglogene autotemcel), developed by CRISPR Therapeutics and Vertex Pharmaceuticals, is the first regulatory-approved cell-based gene therapy for sickle cell disease and beta-thalassemia. - The therapy leverages CRISPR/Cas9 gene-editing technology to deliver a potentially curative, one-time treatment for these inherited blood disorders. - CRISPR Therapeutics reported modest revenue of $11.6 million in the first half of 2026 and a net loss of $214 million, reflecting the high-risk, high-reward nature of gene-editing development. - The company's pipeline extends beyond hemoglobinopathies to include in vivo approaches and regenerative medicine programs.
- Over 30,000 people in the U.S. live with cystic fibrosis, with rising diagnosed prevalence driving demand for targeted therapies and precision medicine approaches. - CFTR modulator therapies have dramatically transformed treatment by targeting underlying CFTR gene mutations, improving lung function, life expectancy, and reducing hospital admissions. - Gene therapy and CRISPR-based gene-editing technologies are emerging as potential long-term or permanent solutions, though they remain in development. - High drug costs and limited treatment options for rare CFTR mutations continue to pose significant barriers to patient access globally.
