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Cystic Fibrosis Foundation

4550 Montgomery Ave. Suite 1100 N, Bethesda, MD, 20814, United States

Overview

The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment and ensuring access to high-quality, specialized care.

Total investments
27
Lead investments
16
Investments · 12mo
1
Active investors
8

Sector focus

  • Association
  • Non Profit
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Investment portfolio

  • Splisense

    Led · Equity · Jun 2026

    SpliSense is a clinical-stage biotech company developing inhaled antisense oligonucleotide therapies and a proprietary inhaled ASO platform designed to modulate gene expression and RNA processing in the lungs. Its lead program, SPL84, is an inhaled ASO therapy in clinical development for cystic fibrosis. The company is advancing a broader pipeline addressing cystic fibrosis, muco-obstructive lung diseases (including COPD, asthma and NCFB) and idiopathic pulmonary fibrosis. SpliSense is led by CEO Gili Hart and is based in Jerusalem, Israel. The recent financing of up to $13M, led by the Cystic Fibrosis Foundation, is earmarked to support continued clinical development of SPL84. The article does not report revenue, user metrics, prior rounds, or valuation information.

  • SNIPR Biome

    Participated · Series B · Aug 2025

    SNIPR BIOME is a clinical-stage biotech in Copenhagen that leverages a novel application of CRISPR-Cas technology to treat and prevent human disease via precision killing of bacteria or gene modification. Its SNIPR technology is applied in collaborations with CARB-X, the Gates Foundation, the Cystic Fibrosis Foundation, IPATH, SPRIN‑D and MD Anderson Cancer Center. The company focuses on microbial CRISPR-medicine, with programs aimed at infectious-disease targets. Led by CEO Christian Grøndahl, SNIPR Biome is advancing CRISPR-based therapeutic approaches toward the clinic. The company recently completed a significant Series B raise, strengthening its balance sheet to continue development. It intends to use the new funds to support a CRISPR-Cas therapy specifically targeting airway infections caused by Pseudomonas aeruginosa in people with cystic fibrosis. SNIPR Biome is a Copenhagen-based clinical-stage biotech pioneering CRISPR-Cas–armed phages (CAPs) as precision medicines to prevent and treat bacterial infections and to modify microbiomes. The company has developed an existing CRISPR-armed phage cocktail, SNIPR001, comprising four CAPs that broadly target Escherichia coli and will leverage this in the new project. SNIPR draws from an extensive phage library with broad antibacterial activity against E. coli and Klebsiella pneumoniae strains sourced from sites in low- and middle-income countries (LMICs). SNIPR was the first company to orally dose humans with a CRISPR therapeutic and holds US and European patents for the use of CRISPR targeting microbiomes. The company works with collaborators including Novo Nordisk, CARB-X, SPRIN-D, and MD Anderson Cancer Center. With new Gates Foundation funding, SNIPR plans to develop microbiome-directed CAPs aimed at reducing gut entero-pathogen burden to improve environmental enteric dysfunction (EED) and pregnancy outcomes in LMICs. SNIPR Biome pioneers precision medicines using CRISPR/Cas for microbial gene therapy, focusing on designer phage and bacteria to edit or kill target microbes. Its lead product, SNIPR001, is a CRISPR-armed phage therapeutic that selectively targets E. coli in the gut and aims to prevent E. coli bloodstream infections in hematological cancer patients. Preclinical data published in Nature Biotechnology showed selective removal of antibiotic-resistant E. coli without off-target effects, and an interim Phase 1 in healthy subjects demonstrated safety, target engagement, and dose-dependent fecal recovery. SNIPR001 has received Fast-Track designation from the FDA for prophylaxis of bloodstream E. coli infections in patients with hematological malignancy at risk of neutropenia. The company collaborates with partners including Novo Nordisk, CARB-X, SPRIN-D, and MD Anderson, and states it was the first to orally dose humans with a CRISPR therapeutic and to obtain US and European patents for CRISPR targeting of microbiomes. The recent funding will support advancing SNIPR001 into patient trials and underpin a further significant fundraise to continue development of its AMR and gut-directed pipeline. SNIPR BIOME ApS is a CRISPR- and microbiome-biotechnology company based in Copenhagen, Denmark. Its lead candidate, SNIPR001, is being developed to prevent Escherichia coli infections in cancer patients, particularly those with hematological malignancies. The program aims to eradicate E. coli that can cause life-threatening bloodstream infections in patients weakened by disease and chemotherapy. SNIPR BIOME is advancing SNIPR001 through preclinical development under an award from CARB-X. The company may receive milestone-based additional funding tied to project progress. The work focuses on an engineered drug approach that leverages the company’s CRISPR and microbiome expertise. Snipr Biome is developing CRISPR-based therapeutics that use bacteria's CRISPR/Cas systems to selectively target and kill bacteria with defined DNA sequences. The company has secured a series of patents on altering microbiota for purposes including immune modulation. Snipr plans to advance its CRISPR microbiome drugs into clinical trials and has been refining its R&D strategy following technology validation. It will initially focus on precision medicines for difficult-to-treat infections and precision microbiome modulation in autoimmunity and cancer. The technology also has potential applications against multi-drug-resistant bacteria and in food-industry quality control. Snipr recently raised $50 million to support these development efforts.

  • Prime Medicine

    Led · Equity · Jul 2025

    Prime Medicine develops therapies using prime editing, a gene editing technology that enables a wide range of DNA modifications with high precision. The company was founded by Drs. David Liu and Andrew Anzalone, pioneers of the prime editing approach. Prime is investigating prime editing for multiple diseases, including cystic fibrosis (CF), and is advancing preclinical work to deliver gene editing cargo to lung cells that produce CFTR. The company is pursuing multiple CF strategies, including a “hotspot” approach for small corrections and PASSIGE for large gene insertions, intended to reduce the need for mutation-specific therapies. Current development is focused on the CF nonsense mutation G542X, a prevalent mutation with no available therapies. Financial support for this work includes prior and additional commitments from the Cystic Fibrosis Foundation to fund preclinical development and delivery research. Prime Medicine develops therapies based on a gene editing technology called prime editing and was founded by researchers who pioneered that approach. The company is investigating whether prime editing could treat several diseases, including cystic fibrosis, by inserting DNA that codes for the CFTR gene. Prime is advancing two platform technologies for CF: “hotspot,” for smaller corrections to specific CFTR mutations, and PASSIGETM, for large gene insertions that could work across many mutations. The company has already used hotspot to correct the G542X nonsense CFTR mutation in the lab and plans to extend that work to other mutation clusters. Prime is also investigating lipid nanoparticle delivery to address challenges delivering genetic therapies to the lungs. The company’s current activities for CF are at the preclinical research stage. Prime Medicine is a Cambridge, MA biotechnology company focused on developing therapies using Prime Editing. Its core platform uses a prime editor protein (a Cas nickase fused to a reverse transcriptase) together with pegRNAs to search for and replace disease‑causing DNA sequences without producing double‑strand breaks. The company is advancing multiple drug discovery programs targeting liver, eye, ex‑vivo hematopoietic stem cell, and neuro‑muscular indications. Leadership includes CEO Keith Gottesdiener, MD; CSO Jeremy Duffield, MD, PhD; and scientific founders David R. Liu, PhD, and Andrew Anzalone, MD, PhD. Prime Medicine intends to use recent funding to progress toward clinical indications and to expand and enhance its platform capabilities. It expects to employ more than 100 full‑time employees as it scales development.

  • ReCode Therapeutics

    Led · Equity · Nov 2024

    ReCode Therapeutics is a clinical-stage biotech developing genetic medicines for cystic fibrosis, leveraging its Selective Organ Targeting lipid nanoparticle delivery platform alongside mRNA and gene-editing approaches. Its lead inhaled program, RCT2100, is in a fully enrolled Phase 2a study and received Fast Track designation from the U.S. FDA in February 2026, with data expected in the fourth quarter. The company has entered a research collaboration with an undisclosed gene-editing company to co-develop therapies that correct CFTR mutations, combining ReCode's delivery technology with the partner's editing platform. The collaboration and funding from the Cystic Fibrosis Foundation are intended to advance one or more candidates toward clinical development and commercialization. Recent leadership changes include Heather Clark being named CEO and former CEO Shehnaaz Suliman becoming executive chair of the board. Clark joined ReCode in 2022 and has extensive rare-disease and cystic fibrosis drug development experience, including more than two decades at Vertex Pharmaceuticals.

  • Sionna Therapeutics

    Participated · Series C · Mar 2024

    Sionna Therapeutics is a clinical-stage life sciences company focused on developing treatments for cystic fibrosis by normalizing the function of the CFTR protein. It is advancing a pipeline of small molecules engineered to correct protein defects caused by the ΔF508 mutation. The company holds a portfolio of programs directly targeting correction of the first nucleotide-binding domain (NBD1) and complementary programs targeting ICL4 and TMD1. Its lead approach aims to fully restore ΔF508-CFTR function by stabilizing NBD1. The company raised a $182M Series C to support clinical development and CEO Mike Cloonan said the financing provides funding through 2026 to execute its clinical plan and deliver multiple readouts. Sionna also added Dr. Fleming to its board, bringing more than 30 years of healthcare industry experience. Sionna Therapeutics is a Boston-based life sciences company developing differentiated small-molecule treatments for cystic fibrosis that aim to normalize CFTR function by stabilizing its first nucleotide-binding domain (NBD1). The company is advancing a pipeline of first-in-class small molecules designed to fully restore CFTR function, including programs targeting NBD1 and complementary modulators of the NBD1–ICL4 interface and TMD1. Sionna plans to submit INDs for its first NBD1-targeted program, SION-638, and for its lead ICL4 program, SION-109, within the next 12 months. The company closed a $111M Series B and has raised approximately $150M to date. Management includes CEO Mike Cloonan, CMO Charlotte McKee, and CSO John Macor, with senior discovery and chemistry leaders Greg Hurlbut and Mark Munson. Its board includes representatives from RA Capital, Atlas Venture, OrbiMed and TPG. The new financing will be used to advance development of the NBD1 franchise and complementary modulators.

Team

  • Wynne Sharples

    Founder

  • Preston Campbell

    Executive Vice President for Medical Affairs

  • Clancy JP

    Vice President of Clinical Research at Cystic Fibrosis

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  • Mike Thompson

    Member, Board of Directors

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