Cystic Fibrosis Foundation
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private
- Established
- 1955-01-01
- Employees
- 501
- Market Cap
- -
- Website
- http://www.cff.org
Prime Medicine Restructures: CEO Replaced, 25% Staff Cut, and Lead Program Halted
• Prime Medicine announced a major restructuring on Monday, replacing its CEO, laying off 25% of staff, and discontinuing its only clinical program despite previous scientific milestones.
• The company's decision reflects broader challenges in the gene editing sector, where numerous firms have reduced workforce or terminated programs during an extended industry downturn.
• This reorganization highlights the disconnect between promising early scientific data and commercial viability in the advanced therapeutic space, particularly for CRISPR-based technologies.
Prime Medicine Reports Breakthrough Clinical Data for First Prime Editing Therapy in Chronic Granulomatous Disease
• Prime Medicine's PM359, the first Prime Editing therapy administered to humans, demonstrated rapid restoration of NADPH oxidase activity in a patient with Chronic Granulomatous Disease, exceeding therapeutic thresholds.
• A single infusion of PM359 achieved 66% DHR positivity by Day 30, significantly above the 20% threshold believed to be potentially curative, with faster engraftment than existing gene editing technologies.
• The therapy showed an encouraging safety profile with no serious adverse events related to PM359, marking a significant milestone for Prime Editing technology as a potential one-time curative treatment for genetic diseases.
Prime Medicine Advances Gene Editing Pipeline with Key Programs for CGD, Wilson's Disease, and AATD
• Prime Medicine expects to release initial clinical data from its Phase 1/2 trial of PM359 for p47phox chronic granulomatous disease in 2025, potentially demonstrating the curative potential of its Prime Editing technology.
• The company recently unveiled its alpha-1 antitrypsin deficiency (AATD) program, which has shown promising preclinical results with high editing efficiency and full restoration of wild-type AAT protein to normal human range.
• Prime Medicine is advancing its Wilson's Disease program (PM577) through IND-enabling studies, with regulatory filings planned for first half of 2026, as part of its expanding liver disease franchise.
Cystic Fibrosis Foundation Invests $2.3 Million in Owlstone Medical to Develop Breath-Based Pathogen Test
• 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 and Galapagos Form $406 Million Alliance to Develop Novel Cystic Fibrosis Therapies
• 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.
Boehringer Ingelheim Launches First-in-Class Inhaled Gene Therapy Trial for Cystic Fibrosis
• Boehringer Ingelheim initiates LENTICLAIR 1 Phase I/II trial evaluating BI 3720931, a novel inhaled gene therapy targeting cystic fibrosis using Oxford Biomedica's lentiviral vector technology.
• The innovative therapy aims to address the 10-15% of cystic fibrosis patients who cannot benefit from current CFTR modulators due to mutation type or intolerance.
• Oxford Biomedica's proprietary lentiviral vector manufacturing expertise will support the development of this groundbreaking treatment that delivers functional CFTR genes to airway epithelial cells.
4DMT Prioritizes 4D-150 and 4D-710, Extends Cash Runway into 2028
• 4DMT is focusing its pipeline on 4D-150 for wet AMD and DME and 4D-710 for cystic fibrosis, leveraging their strong clinical proof of concept.
• Phase 3 trials (4FRONT-1 and 4FRONT-2) for 4D-150 in wet AMD are set to begin in Q1 and Q3 2025, respectively, with topline data expected in H2 2027.
• The company's cash runway has been extended to 2028, supported by $506M in cash reserves and strategic resource allocation.
• 4DMT will discontinue development of early-stage programs like 4D-110 for choroideremia and 4D-125 for X-linked retinitis pigmentosa.
Vertex's ALYFTREK Approved by FDA as Next-Generation Cystic Fibrosis Treatment
• The FDA has approved Vertex's ALYFTREK for cystic fibrosis (CF) in patients aged 6 years and older with specific genetic mutations.
• ALYFTREK is a once-daily triple combination therapy, offering a more convenient dosing schedule compared to existing treatments.
• Clinical trials demonstrated ALYFTREK's non-inferiority to TRIKAFTA in lung function and significant improvement in sweat chloride levels.
• The drug's label includes a boxed warning for potential liver injury, necessitating regular liver function monitoring.
Pfizer Expands Drug Discovery Alliance with Two More Flagship Startups for Cancer and Obesity Research
• Pfizer has partnered with Flagship Pioneering's Montai Therapeutics to develop novel small molecule drugs for lung cancer, leveraging AI-driven discovery platforms and shared R&D costs.
• Ampersand Biomedicines joins the alliance to develop tissue-selective biologics for obesity treatment, building on their $50 million-backed platform technology.
• These partnerships are part of Pfizer's broader 2023 alliance with Flagship Pioneering to discover up to 10 new drug candidates, following earlier deals with ProFound and Quotient Therapeutics.
NIH Awards $2.9 Million to ACRI for Cystic Fibrosis Therapy Research
• The NIH has granted $2.9 million to ACRI to study how to improve the effectiveness of a key therapy for cystic fibrosis (CF).
• The five-year study will investigate optimal dosing and potential genetic factors influencing response to triple combination therapy in CF patients.
• Researchers aim to personalize CF treatment by understanding how individual patients process the drug and identifying the best dose for each person.
• The study seeks to address the challenges of variable treatment response and side effects associated with triple combination therapy in CF patients.
Improved Medications and Comprehensive Care Transform Life for Cystic Fibrosis Patient
• Chrystal Moldenhauer, diagnosed with cystic fibrosis (CF) at three months old, has experienced a significant improvement in her quality of life due to advances in medicine and comprehensive care at Sanford Health.
• Effective medications, including Kalydeco, have reduced the need for extensive daily treatments, allowing Chrystal to engage more fully in family life and other activities.
• The median age at death for people with CF was 66 years in 2022, and their life expectancy was only 12% lower than that of healthy people.
• Chrystal's care team emphasizes the importance of patient diligence and participation in drug trials for the continued development of new and improved CF treatments.
Arcturus Therapeutics Gains Momentum with Regulatory Approval and Clinical Advancements
• Arcturus Therapeutics' Kostaive vaccine receives Japanese regulatory approval for updated JN.1 formulation, expected to generate $85 million in revenue.
• The company's cystic fibrosis treatment, ARCT-032, advances to Phase 2 study after promising Phase 1/1b trial results.
• Arcturus is awaiting European Medicines Agency (EMA) decision on Kostaive approval, anticipated in the third quarter of 2024.
RDCA-DAP Boosts Cystic Fibrosis Research with New Data Sets
• The RDCA-DAP has acquired new datasets from the Cystic Fibrosis Therapeutics Development Network to accelerate cystic fibrosis (CF) research and therapy development.
• The data sharing initiative seeks to enhance understanding of CF progression, design new clinical outcome measures and biomarkers, and develop disease models.
• By integrating data into the RDCA-DAP, researchers worldwide can access and leverage data, potentially leading to breakthroughs in CF care.
• The RDCA-DAP is an initiative run by the Critical Path Institute (C-Path) and funded by the FDA to provide a centralized platform of patient data for rare diseases.
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