Cystic Fibrosis Foundation

Developer of life sciences technology intended to develop highly effective and differentiated treatments for cystic fibrosis. The company is developing first-in-class small molecules that are focused on a unique target with the potential to fully normalize CFTR function for the first time for the vast majority of people living with cystic fibrosis, enabling patients with best-in-class efficacy and reach and long-term benefit for people with cystic fibrosis.

Eloxx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing novel ribonucleic acid modulating drug candidates for the treatment of rare and ultra-rare premature stop codon diseases. Its lead investigational drug product candidate is ELX-02, which is in Phase 2 clinical trial that focuses on the treatment of cystic fibrosis and nephropathic cystinosis patients with diagnosed nonsense mutations. The company was founded in 2013 and is headquartered in Waltham, Massachusetts.

Feldan Bio Inc. is a recombinant protein company based in Quebec, Canada, specializing in the discovery, development, manufacture, and marketing of recombinant proteins and molecular biology reagents for life science companies and research laboratories globally. The company has developed the Feldan Shuttle, a patented protein-based technology platform that facilitates the delivery of transcription factors and genome editing tools for clinical applications. Feldan Bio offers a wide range of products, including monoclonal antibodies, cell culture supplements, transfection reagents, and various growth factors and enzymes. Additionally, it provides research protein products and custom services such as protein expression, antibody generation, and custom peptide synthesis. The company also develops and licenses therapeutic proteins aimed at biopharmaceutical companies focusing on treatments for diseases like cancer and diabetes. Founded in 1999, Feldan Bio is committed to advancing therapeutic applications through its innovative technology platform.

SalioGen Therapeutics advances in curative genetic therapies using its Exact DNA Integration Technology (EDIT) platform, a mammal-derived genome engineering technology. It is focused on providing durable, safe, and affordable non-viral gene therapies to more patients with inherited diseases. Looking ahead, SalioGen will also explore the EDIT platform’s capabilities to streamline cell therapy, cell engineering, and biologics manufacturing to help an even broader population of patients in need.

Eloxx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing novel ribonucleic acid modulating drug candidates for the treatment of rare and ultra-rare premature stop codon diseases. Its lead investigational drug product candidate is ELX-02, which is in Phase 2 clinical trial that focuses on the treatment of cystic fibrosis and nephropathic cystinosis patients with diagnosed nonsense mutations. The company was founded in 2013 and is headquartered in Waltham, Massachusetts.

Hunterian Medicine is a biotech company that develops cures for a vast number of genetic diseases where no FDA approved therapies exist. It is poised to unleash the full potential of CRISPR. Its patented platform technology solves the CRISPR delivery problem by enabling efficient, on-target delivery through a single adeno-associated virus (AAV). The Company was founded in 2016 and is headquartered in Cambridge, Massachusetts.

Metagenomi, Inc. is a genetic medicines company based in Emeryville, California, focused on developing innovative gene editing systems for the treatment of genetic diseases. Since its incorporation in 2016, the company has utilized a proprietary metagenomics-derived genome editing toolbox that enables the creation of curative therapeutics. This toolbox encompasses a range of advanced technologies, including programmable nucleases, base editors, and various RNA and DNA-mediated integration systems, such as prime editing systems and CRISPR-associated transposases (CASTs). Metagenomi aims to address the challenges posed by diverse mutations that have traditionally been difficult to target with existing genome engineering approaches.

Splisense is develops antisense oligonucleotide based therapies to target genetic diseases caused by splicing mutations. The company has developed a compound to cure the lung disease in cystic fibrosis patients carrying a specific splicing mutation. It is also developing a targeted modulation approach to correct the splicing pattern of CFTR transcripts according to the specific mutation carried.

Eloxx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing novel ribonucleic acid modulating drug candidates for the treatment of rare and ultra-rare premature stop codon diseases. Its lead investigational drug product candidate is ELX-02, which is in Phase 2 clinical trial that focuses on the treatment of cystic fibrosis and nephropathic cystinosis patients with diagnosed nonsense mutations. The company was founded in 2013 and is headquartered in Waltham, Massachusetts.

enGene, Inc. is a biotechnology company specializing in mucosal immunotherapy platforms aimed at treating inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables localized delivery of immune-modulating proteins to mucosal tissues, including the gastrointestinal tract, lung, and bladder. This innovative approach allows for the treatment of various immune disorders and supports systemic release of proteins from the gut to address conditions such as diabetes, anemia, and hemophilia. Founded in 1999 and based in Vancouver, Canada, enGene has formed a strategic alliance with Takeda Pharmaceutical Company Ltd. The company's platform facilitates the induction or suppression of protein expression levels, which can help regenerate physiologic, meal-regulated insulin secretion for diabetes patients.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing targeted gene therapy products using evolved adeno-associated virus vectors. The company has a diverse portfolio focusing on three main therapeutic areas: ophthalmology, cardiology, and pulmonology. Key product candidates include 4D-125, currently in a Phase I/II clinical trial for treating X-linked retinitis pigmentosa; 4D-110, in a Phase I trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is developing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. The company aims to address unmet medical needs through its innovative gene therapies.

Eloxx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing novel ribonucleic acid modulating drug candidates for the treatment of rare and ultra-rare premature stop codon diseases. Its lead investigational drug product candidate is ELX-02, which is in Phase 2 clinical trial that focuses on the treatment of cystic fibrosis and nephropathic cystinosis patients with diagnosed nonsense mutations. The company was founded in 2013 and is headquartered in Waltham, Massachusetts.

Talee Bio is a rapidly growing gene therapy company developing the next generation treatment and cures for cystic fibrosis.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing targeted gene therapy products using evolved adeno-associated virus vectors. The company has a diverse portfolio focusing on three main therapeutic areas: ophthalmology, cardiology, and pulmonology. Key product candidates include 4D-125, currently in a Phase I/II clinical trial for treating X-linked retinitis pigmentosa; 4D-110, in a Phase I trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is developing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. The company aims to address unmet medical needs through its innovative gene therapies.