相关临床试验
3
2 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
1955
已完成
1
33.3%
尚未招募
2
66.7%
暂无批准数据
- 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.
- SpliSense entered a funding agreement with the Cystic Fibrosis Foundation for up to $13 million to support the Phase 2b clinical development of SPL84, an inhaled antisense oligonucleotide therapy for cystic fibrosis. - The funding follows positive Phase 2a results showing lung function improvement in up to 70% of treated participants and an estimated mean absolute improvement of 10 percentage points in ppFEV1 versus placebo. - The ongoing randomized, placebo-controlled Phase 2b study will enroll approximately 40 participants across the U.S., Europe, and Israel, with topline results expected in H2 2027. - SPL84 targets the 3849+10kb C→T splicing mutation in the CFTR gene and has received FDA Fast Track and Orphan Drug designations as well as EMA PRIME designation.
- Blackstone Life Sciences has invested $250 million in Anagram Therapeutics to advance ANG003, a novel oral enzyme replacement therapy for exocrine pancreatic insufficiency in cystic fibrosis patients. - ANG003 represents a significant advancement by requiring only one tablet per meal compared to the current standard of up to 40 pills daily, potentially improving patient compliance and quality of life. - The therapy is the first non-porcine enzyme replacement product, addressing global supply shortages of existing pig-derived treatments in a market worth approximately $2 billion annually. - Anagram plans to initiate an international Phase 2 study following positive results from earlier clinical trials in patients with exocrine pancreatic insufficiency due to cystic fibrosis.
- Antiverse closed a $9.3 million Series A financing led by Soulmates Ventures to accelerate its AI-driven antibody design platform and advance therapeutic programs toward in vivo efficacy studies. - The company entered a research agreement with the Cystic Fibrosis Foundation to design novel antibodies targeting the extracellular region of CFTR protein, a historically difficult target in cystic fibrosis research. - Antiverse's proprietary platform can reduce development time for de novo therapeutic-grade antibodies to under four months, positioning the company to tackle challenging drug targets including G-protein coupled receptors and ion channels. - The funding brings total investment to over $20 million since inception and will support expansion of pharmaceutical collaborations and progression of wholly owned candidates into preclinical development by 2027.
- The Cystic Fibrosis Foundation will provide up to $11 million in additional funding to 4D Molecular Therapeutics to accelerate development of 4D-710, a gene therapy for cystic fibrosis lung disease. - Phase 2 enrollment in the AEROW clinical trial is currently underway with 2.5E14 vg selected as the anticipated pivotal and commercial dose for the variant-agnostic genetic medicine. - The funding supports redosing studies and Phase 3 readiness, with interim Phase 1 data including functional durability results expected by year-end 2025. - 4D-710 represents the first known genetic medicine to demonstrate successful CFTR transgene delivery and expression throughout CF patient airways via aerosol delivery.
- ReCode Therapeutics raised over $29 million in additional financing to advance its genetic medicines pipeline, including investigational therapies for cystic fibrosis. - The Cystic Fibrosis Foundation committed an additional $3 million to support the ongoing Phase 2 clinical trial of RCT2100, bringing total CF Foundation investment to up to $33 million. - RCT2100 is designed to deliver functional CFTR protein via inhaled mRNA therapy, potentially benefiting all CF patients including those with rare mutations who don't respond to existing modulator therapies. - The company also announced a research collaboration with Praxis Precision Medicines to develop lipid nanoparticle formulations for enhanced antisense oligonucleotide delivery to brain regions.
- The Cystic Fibrosis Foundation has committed up to $24 million in additional funding to Prime Medicine to advance prime editing gene therapy for cystic fibrosis patients. - The funding will focus on developing a prime editing therapy targeting the G542X nonsense mutation, one of the most prevalent CF-causing mutations with no available treatments. - Prime Medicine's prime editing technology has the potential to permanently correct CFTR gene mutations and could eventually treat the vast majority of people with cystic fibrosis. - The company is investigating multiple approaches including hotspot editing and PASSIGE technology to address various CF mutations through a single platform.
- Owlstone Medical has secured a $2.3 million equity investment from the Cystic Fibrosis Foundation to develop a breath test for detecting Pseudomonas aeruginosa infections in CF patients. - The breath-based diagnostic aims to match or exceed the accuracy of traditional sputum culture tests while offering the ability to distinguish P. aeruginosa from other common respiratory pathogens like S. aureus. - Initial research will focus on chronic P. aeruginosa infections to establish proof-of-principle, with potential expansion to early detection capabilities—critical for improving outcomes in CF patient care.
• AbbVie has partnered with Galapagos NV in a global alliance worth up to $406 million to discover and develop combination therapies targeting key mutations in cystic fibrosis patients. • The collaboration aims to develop oral drugs addressing F508del and G551D mutations in the CFTR protein, with plans to initiate Phase 1 clinical trials by the end of 2014. • This partnership joins other significant industry efforts to combat cystic fibrosis, including Pfizer's expanded six-year research program with the Cystic Fibrosis Foundation worth up to $58 million.
• The Cystic Fibrosis Foundation is investing $15 million in Recode Therapeutics to advance gene-editing medicines for cystic fibrosis (CF) patients without effective treatment options. • Recode Therapeutics collaborates with Intellia Therapeutics, developing a strategy to alter the genetic code, creating a healthy CFTR gene version for functional protein production. • The funding supports the development of up to three gene-correction programs, initially focusing on CF mutations not addressed by existing therapies. • Recode's RCT2100, an inhaled mRNA therapy delivering a healthy CFTR version to lung cells, is currently in Phase 1 clinical trials, expanding treatment options for all CF patients.
