Cystic Fibrosis Foundation

Owlstone Medical develops and commercializes breathalyzers for clinical diagnostics and precision medicine. Its products detect volatile organic compounds in breath samples to diagnose diseases like lung cancer, asthma, and tuberculosis. The company has strategic collaborations with pharmaceutical companies and is based in Cambridge, UK.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its innovative Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aimed at restoring normal genetic function. The company targets a wide range of diseases with significant unmet medical needs, leveraging the efficiency and versatility of its gene editing capabilities to address the underlying causes of these conditions. Through its commitment to advancing gene editing, Prime Medicine seeks to transform the landscape of genetic medicine.

Clarametyx Biosciences, Inc. is a biotechnology company founded in 2020 and headquartered in Columbus, Ohio. The company focuses on developing targeted and immune-enabling biologic therapies that address persistent infections associated with biofilms, which are protective layers that shield bacteria and complicate treatment. Clarametyx has created a novel technology platform that allows for the targeting of these biofilms, thereby enhancing immune response and facilitating antibiotic intervention. One of its key products in development is CMTX-001, a humanized monoclonal antibody currently in the late preclinical stage, aimed at treating hospital-acquired pneumonia. Clarametyx Biosciences is dedicated to combating life-threatening infections and reducing antibiotic resistance through its innovative therapeutic solutions.

ViaNautis specializes in the development of innovative medications that effectively cross biological barriers, including the blood-brain barrier. The company employs its proprietary PolyNaut technology, a versatile nano-engineered polymer system designed for intracellular delivery. This technology allows for the encapsulation of various therapeutic modalities into polymer nanovesicles, which can selectively target different tissues and cell types. By facilitating the penetration of these barriers, ViaNautis enhances the therapeutic efficacy of the encapsulated molecules, providing significant benefits in medication delivery and treatment outcomes. Collaborating with multiple partners, the company aims to revolutionize how medications are developed and delivered, particularly in addressing challenging medical conditions.

Founded in 2013, Arcturus Therapeutics is a biotechnology company focused on developing RNA medicines for rare diseases, with a current emphasis on liver and respiratory conditions. The company owns proprietary technologies such as UNA chemistry and LUNAR platform, enabling it to target any gene in the human genome.

Nanite is a biotechnology company focused on non-viral gene delivery through the development of programmable polymer nanoparticles. Utilizing an artificial intelligence-driven platform, Nanite integrates advanced high-throughput experimental and computational techniques to create tailored delivery vehicles capable of transporting a diverse array of genetic materials with specific targeting capabilities. This innovative approach aims to assist scientists in functional chemistry by providing therapeutically relevant solutions for gene delivery, enhancing the potential for various medical applications and indications.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Gensaic is a biotechnology company developing a machine-guided protein design platform. This platform aims to create tissue-selective, intracellular therapies for metabolic and age-related diseases, with a focus on muscle-wasting and obesity.

Aridis Pharmaceuticals is a late-stage biopharmaceutical company based in San Jose, California, founded in 2003. The company specializes in discovering and developing targeted immunotherapy treatments using fully human monoclonal antibodies (mAbs) to combat life-threatening infections. Its lead product candidate, AR-301, is currently in Phase III trials for treating lung infections caused by S. aureus alphatoxin. Additionally, Aridis Pharmaceuticals is advancing several other mAb candidates through various stages of clinical development, including AR-105 and AR-101 for gram-negative bacterial infections, AR-401 for Acinetobacter baumannii infections, AR-201 for respiratory syncytial virus, and AR-501 for chronic lung infections in cystic fibrosis patients. The company employs proprietary technology platforms to rapidly identify and manufacture these mAbs from patients who have successfully overcome infections, leveraging the natural potency of the human immune system.

Founded in London in 2007, Pulmocide specializes in developing inhaled medicines targeting serious viral and fungal respiratory infections. Its focus is on creating drugs optimized for lung delivery to maximize efficacy while minimizing systemic exposure.

Carmine Therapeutics, established in 2019 and headquartered in Singapore, specializes in gene therapy. It produces extracellular vesicles from red blood cells as a non-viral, re-dosable gene delivery vehicle, addressing challenges posed by traditional viral-based therapies such as immunogenicity, limited transgene capacity, and manufacturing complexities. The company focuses on developing next-generation gene therapies for hematology, oncology, and immunology.

Destiny Pharma is a clinical-stage biotechnology company based in Brighton, United Kingdom, specializing in the discovery and development of innovative antimicrobial products aimed at treating infectious diseases. The company's lead candidates include XF-73 Nasal, currently in Phase II clinical trials for the prevention of post-surgical staphylococcal infections, and XF-73 Dermal, in Phase I trials for treating skin infections caused by antibiotic-resistant bacteria. Destiny Pharma also develops other compounds, such as XF-70 for treating ventilator-associated pneumonia and DPD-207 for eye infections, as well as additional XF drugs targeting antibiotic-resistant biofilm infections. The company has established several collaboration agreements with various academic institutions and organizations, enhancing its research capabilities and broadening its therapeutic focus. Founded in 1996, Destiny Pharma is committed to addressing the growing global healthcare challenge posed by drug-resistant bacteria.

Carbon Biosciences specializes in developing genetic medicines for treating severe diseases. They employ industrial-scale viral production platforms to create vectors capable of delivering diverse therapeutic modalities.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, specializing in the development of novel drugs targeting rare and ultra-rare genetic diseases caused by nonsense mutations. Founded in 2013, the company is focused on discovering and commercializing ribonucleic acid modulating drug candidates. Its lead investigational product, ELX-02, is currently undergoing Phase 2 clinical trials aimed at treating cystic fibrosis and nephropathic cystinosis in patients with specific nonsense mutations. Eloxx utilizes its advanced technology platform to create oral small molecules that can induce ribosomal readthrough, thereby enabling the production of full-length functional proteins. The company is building a robust pipeline of therapeutic candidates designed to address various genetic disorders, including Duchenne Muscular Dystrophy and Tay-Sachs syndrome, by overcoming the limitations posed by premature stop codons.

Felix Biotechnology, Inc. is a San Francisco-based company founded in 2019 that specializes in developing biotherapeutics to combat microbial challenges in human health. The company focuses on creating treatments for antibiotic-resistant strains of bacteria and fungi, aiming to address the urgent need for effective management of infectious diseases. By accelerating the deployment of novel biotherapeutics, Felix Biotechnology seeks to provide healthcare professionals with precise and effective tools to tackle these pressing health issues.

Feldan Therapeutics specializes in intracellular drug delivery using its patented Feldan Shuttle technology. This platform enables rapid, safe, and efficient transport of therapeutic agents into cells, with a focus on developing treatments for serious diseases such as skin and lung conditions.

SalioGen Therapeutics develops curative therapies for inherited disorders using its Exact DNA Integration Technology (EDIT) platform. EDIT employs proprietary mammal-derived enzymes called Saliogase, enabling non-viral delivery of genes of any size and offering significant manufacturing advantages.

Peptilogics, Inc. is a clinical-stage biotechnology company based in West Mifflin, Pennsylvania, founded in 2013. The company specializes in designing and developing novel peptide therapeutics aimed at addressing the urgent challenge of multidrug-resistant bacterial infections. Peptilogics utilizes a unique computational platform that enhances drug discovery by exploring new chemical design spaces while ensuring the biological viability and manufacturability of potential therapeutics. Their lead candidate, PLG0206, is a broad-spectrum anti-infective peptide that targets prosthetic joint infections and has received both FDA Orphan Drug Designation and Qualified Infectious Disease Product Designation. Through its innovative eCAP (engineered cationic antibiotic peptide) platform, Peptilogics aims to provide effective treatment options against resistant bacteria and biofilms, ultimately improving patient outcomes and affordability in healthcare.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

EnBiotix, Inc. is a bioengineering company that specializes in developing therapies for chronic and recurrent pulmonary infections, particularly those caused by drug-resistant bacteria. Founded in 2010 and headquartered in Boston, Massachusetts, the company employs innovative platforms, including an anti-persisters platform that enhances antibiotic effectiveness, an engineered bacteriophage platform that targets infections, and a tunable target degradation platform that modifies protein functions. EnBiotix has a diverse clinical pipeline, with notable products such as EBX-001 for P. aeruginosa infections in cystic fibrosis patients, EBX-002 for catheter-associated urinary tract infections, and EPP-001 for prosthetic joint infections. The company's mission is to combat the global rise of antibiotic resistance by revitalizing existing antibiotic classes and addressing a significant market need for effective treatments in the healthcare landscape.

Founded in London in 2007, Pulmocide specializes in developing inhaled medicines targeting serious viral and fungal respiratory infections. Its focus is on creating drugs optimized for lung delivery to maximize efficacy while minimizing systemic exposure.

Armata Pharmaceuticals is a clinical-stage biotechnology company headquartered in Marina del Rey, California, specializing in the development of targeted bacteriophage therapeutics for addressing drug-resistant bacterial infections. Founded in 2005, the company aims to combat the rising threat of multidrug-resistant bacteria, which has been exacerbated by antibiotic overuse and poses significant health risks. Armata focuses on creating pathogen-specific bacteriophage therapies, leveraging both natural and synthetic phage candidates to effectively treat infections caused by bacteria such as Pseudomonas aeruginosa and Staphylococcus aureus. The company is advancing a diverse pipeline of therapeutic candidates and is collaborating with Merck to develop proprietary synthetic phage therapies for specific infectious disease agents.

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, specializing in the development of novel drugs targeting rare and ultra-rare genetic diseases caused by nonsense mutations. Founded in 2013, the company is focused on discovering and commercializing ribonucleic acid modulating drug candidates. Its lead investigational product, ELX-02, is currently undergoing Phase 2 clinical trials aimed at treating cystic fibrosis and nephropathic cystinosis in patients with specific nonsense mutations. Eloxx utilizes its advanced technology platform to create oral small molecules that can induce ribosomal readthrough, thereby enabling the production of full-length functional proteins. The company is building a robust pipeline of therapeutic candidates designed to address various genetic disorders, including Duchenne Muscular Dystrophy and Tay-Sachs syndrome, by overcoming the limitations posed by premature stop codons.

Metagenomi, Inc. is a genetic medicines company focused on developing innovative gene editing systems to address genetic diseases. Incorporated in 2016 and headquartered in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to discover novel genome editing technologies. By sourcing cellular machinery from previously uncharacterized organisms, the company creates a proprietary toolbox that includes programmable nucleases, base editors, and advanced integration systems like prime editing and CRISPR-associated transposases. These systems are designed to be ultra-small, efficient, and highly specific, minimizing the risk of immune responses. Metagenomi aims to revolutionize gene editing techniques and deliver curative therapeutics to patients worldwide.

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.

Microbion Corporation is a biopharmaceutical company dedicated to developing products for treating resistant and difficult-to-treat infections. It specializes in bismuth-thiols, used to prevent microbial biofilms on medical devices and manage infectious diseases. Founded in 1982, the company is headquartered in Bozeman, Montana.

Kinnear Pharmaceuticals is a preclinical stage pharmaceutical company committed to the discovery, development and commercialization of ceragenin-based anti-infective and anti-inflammatory product candidates for diseases with large unmet clinical needs.

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, specializing in the development of novel drugs targeting rare and ultra-rare genetic diseases caused by nonsense mutations. Founded in 2013, the company is focused on discovering and commercializing ribonucleic acid modulating drug candidates. Its lead investigational product, ELX-02, is currently undergoing Phase 2 clinical trials aimed at treating cystic fibrosis and nephropathic cystinosis in patients with specific nonsense mutations. Eloxx utilizes its advanced technology platform to create oral small molecules that can induce ribosomal readthrough, thereby enabling the production of full-length functional proteins. The company is building a robust pipeline of therapeutic candidates designed to address various genetic disorders, including Duchenne Muscular Dystrophy and Tay-Sachs syndrome, by overcoming the limitations posed by premature stop codons.

Spexis AG is a clinical-stage biopharmaceutical company based in Allschwil, Switzerland, specializing in the discovery and development of macrocycle drugs and antibiotics to address severe or life-threatening diseases. Founded in 1996, the company focuses on addressing high unmet medical needs, particularly in the areas of antibiotic resistance and respiratory diseases. Spexis's lead product, murepavadin, is currently in Phase III development targeting Pseudomonas aeruginosa, a pathogen identified as a critical priority by the World Health Organization. The company's operations are centered on research, development, and the prospective commercialization of innovative therapeutics in the respiratory domain.

Calithera Biosciences, Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule drugs targeting tumor metabolism and immunology for cancer treatment. Headquartered in South San Francisco, California, the company’s lead product candidate, CB-839, is an inhibitor of glutaminase currently in Phase II clinical trials for solid tumors. Additionally, Calithera is developing INCB001158, an oral arginase inhibitor in Phase I/II trials for hematology and oncology, as well as CB-280, another oral arginase inhibitor aimed at treating cystic fibrosis, and CB-708, an orally administered small molecule inhibitor of CD73. The company has established collaborations with Bristol-Myers Squibb to evaluate the combination of nivolumab with CB-839, and with Incyte Corporation for the development and commercialization of INCB001158. Founded in 2010, Calithera Biosciences is dedicated to advancing innovative therapies that specifically target critical pathways in tumor growth and survival.

EnGene specializes in developing a mucosal immunotherapy platform for treating inflammatory bowel disease and diabetes. Its core technology enables localized delivery of immune-modulating proteins to intestinal mucosa using non-integrating biopolymer-based nucleotides, impacting diseases affecting mucosal tissues and facilitating systemic protein release.

Synspira Therapeutics is focused on developing a rationally designed portfolio of products in cystic fibrosis and other rare diseases. Inspired by patients and frustrated by the limitations of conventional treatments, Synspira is advancing two novel products (SNSP113 and SNSP003) in order to provide better clinical outcomes and reduce treatment burden in Progressive Pulmonary Disease and Malabsorption Syndromes.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Microbion Corporation is a biopharmaceutical company dedicated to developing products for treating resistant and difficult-to-treat infections. It specializes in bismuth-thiols, used to prevent microbial biofilms on medical devices and manage infectious diseases. Founded in 1982, the company is headquartered in Bozeman, Montana.

Crestone is a biopharmaceutical company focused on developing novel therapies for serious bacterial infections. Its pipeline includes treatments for C. difficile infection, antibiotic-resistant Gram-positive infections, and tuberculosis.

Laurent Pharmaceuticals Inc., established in 2012 and based in Montreal, Canada, is a biopharmaceutical company focused on developing orally-active drug candidates to modulate the immuno-inflammatory response in cystic fibrosis patients. Its lead product, LAU-7b, is a novel once-a-day oral form of fenretinide currently in clinical development stages.

Enterprise Therapeutics is a United Kingdom-based drug discovery company founded in 2014 and based in Brighton. It develops novel disease-modifying therapies for respiratory diseases, including cystic fibrosis, asthma, and COPD, with a focus on addressing the underlying mechanisms of mucus congestion to improve breathing, reduce lung infections, and enhance patient quality of life.

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, specializing in the development of novel drugs targeting rare and ultra-rare genetic diseases caused by nonsense mutations. Founded in 2013, the company is focused on discovering and commercializing ribonucleic acid modulating drug candidates. Its lead investigational product, ELX-02, is currently undergoing Phase 2 clinical trials aimed at treating cystic fibrosis and nephropathic cystinosis in patients with specific nonsense mutations. Eloxx utilizes its advanced technology platform to create oral small molecules that can induce ribosomal readthrough, thereby enabling the production of full-length functional proteins. The company is building a robust pipeline of therapeutic candidates designed to address various genetic disorders, including Duchenne Muscular Dystrophy and Tay-Sachs syndrome, by overcoming the limitations posed by premature stop codons.

Celtaxsys, Inc. is a clinical-stage biopharmaceutical company based in Atlanta, Georgia, founded in 2007. The company specializes in the discovery and development of novel therapeutics aimed at treating inflammatory diseases, including cystic fibrosis and moderately severe acne. Its lead product, CTX-4430, is a small-molecule drug that targets the enzyme Leukotriene A4 hydrolase in immune cells. Celtaxsys also focuses on manipulating immune cell migration and activation to develop treatments for cancer, inflammatory conditions, and autoimmune diseases. Through its innovative approach, the company aims to provide effective therapies for patients suffering from rare and serious inflammatory disorders.

Spirovant Sciences, Inc., established in 2016 and headquartered in Philadelphia, Pennsylvania, specializes in the development of gene therapies for cystic fibrosis and related pulmonary diseases. The company aims to overcome historical barriers in genetic treatments, focusing on adeno-associated viral and lentiviral gene therapy platforms to deliver large payloads, thereby enhancing therapeutic potential. As of late 2019, Spirovant operates as a subsidiary of Sumitovant Biopharma Ltd.

Vast Therapeutics, Inc. is a preclinical-stage pharmaceutical company based in Durham, North Carolina, focused on developing inhalable treatments for antibiotic-resistant pathogens affecting patients with cystic fibrosis and other respiratory diseases. Established in 2017 and previously known as Novoclem Therapeutics, the company aims to address chronic infections that are prevalent in the cystic fibrosis population. Vast Therapeutics operates as a subsidiary of KNOW Bio, LLC, and is dedicated to advancing its innovative therapeutic solutions to improve health outcomes for patients facing these challenging conditions.

Aridis Pharmaceuticals is a late-stage biopharmaceutical company based in San Jose, California, founded in 2003. The company specializes in discovering and developing targeted immunotherapy treatments using fully human monoclonal antibodies (mAbs) to combat life-threatening infections. Its lead product candidate, AR-301, is currently in Phase III trials for treating lung infections caused by S. aureus alphatoxin. Additionally, Aridis Pharmaceuticals is advancing several other mAb candidates through various stages of clinical development, including AR-105 and AR-101 for gram-negative bacterial infections, AR-401 for Acinetobacter baumannii infections, AR-201 for respiratory syncytial virus, and AR-501 for chronic lung infections in cystic fibrosis patients. The company employs proprietary technology platforms to rapidly identify and manufacture these mAbs from patients who have successfully overcome infections, leveraging the natural potency of the human immune system.

Laurent Pharmaceuticals Inc., established in 2012 and based in Montreal, Canada, is a biopharmaceutical company focused on developing orally-active drug candidates to modulate the immuno-inflammatory response in cystic fibrosis patients. Its lead product, LAU-7b, is a novel once-a-day oral form of fenretinide currently in clinical development stages.

Icagen, Inc. is a biopharmaceutical company based in Durham, North Carolina, specializing in drug discovery with a particular emphasis on neuroscience and rare diseases. Founded in 2003, the company focuses on developing novel orally-administered small molecule drugs that target ion channels, among other therapeutic areas. Icagen provides a range of pre-clinical drug discovery services, including assay development, cell line generation, high-throughput screening, medicinal and computational chemistry, and custom assay services. The company's drug pipeline includes ICA-105665, currently in Phase I trials for epilepsy and neuropathic pain, and Senicapoc, which is undergoing Phase I studies for asthma. Icagen actively collaborates with major pharmaceutical companies, such as Bristol-Myers Squibb, Astellas Pharma, and Pfizer, to advance its research and development initiatives.

Laurent Pharmaceuticals Inc., established in 2012 and based in Montreal, Canada, is a biopharmaceutical company focused on developing orally-active drug candidates to modulate the immuno-inflammatory response in cystic fibrosis patients. Its lead product, LAU-7b, is a novel once-a-day oral form of fenretinide currently in clinical development stages.

Synspira Therapeutics is focused on developing a rationally designed portfolio of products in cystic fibrosis and other rare diseases. Inspired by patients and frustrated by the limitations of conventional treatments, Synspira is advancing two novel products (SNSP113 and SNSP003) in order to provide better clinical outcomes and reduce treatment burden in Progressive Pulmonary Disease and Malabsorption Syndromes.

Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP, in Phase 2a development for nontuberculous mycobacteria, or NTM, lung infection, and in preparation for Phase 2a development in cystic fibrosis, or CF, affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in CF. Savara’s strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2 ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Founded in 2001, Sound Pharmaceuticals is a biopharmaceutical company based in Seattle, Washington. It focuses on developing drug treatments for sensorineural hearing loss by targeting growth inhibitory proteins to stimulate the division of inner ear epithelial cells.

Aridis Pharmaceuticals is a late-stage biopharmaceutical company based in San Jose, California, founded in 2003. The company specializes in discovering and developing targeted immunotherapy treatments using fully human monoclonal antibodies (mAbs) to combat life-threatening infections. Its lead product candidate, AR-301, is currently in Phase III trials for treating lung infections caused by S. aureus alphatoxin. Additionally, Aridis Pharmaceuticals is advancing several other mAb candidates through various stages of clinical development, including AR-105 and AR-101 for gram-negative bacterial infections, AR-401 for Acinetobacter baumannii infections, AR-201 for respiratory syncytial virus, and AR-501 for chronic lung infections in cystic fibrosis patients. The company employs proprietary technology platforms to rapidly identify and manufacture these mAbs from patients who have successfully overcome infections, leveraging the natural potency of the human immune system.

Laurent Pharmaceuticals Inc., established in 2012 and based in Montreal, Canada, is a biopharmaceutical company focused on developing orally-active drug candidates to modulate the immuno-inflammatory response in cystic fibrosis patients. Its lead product, LAU-7b, is a novel once-a-day oral form of fenretinide currently in clinical development stages.

Laurent Pharmaceuticals Inc., established in 2012 and based in Montreal, Canada, is a biopharmaceutical company focused on developing orally-active drug candidates to modulate the immuno-inflammatory response in cystic fibrosis patients. Its lead product, LAU-7b, is a novel once-a-day oral form of fenretinide currently in clinical development stages.

Founded in 2009, Corbus Pharmaceuticals is a clinical-stage company focused on developing and commercializing therapeutics for rare, chronic, and serious inflammatory and fibrotic diseases. Its lead product candidate, lenabasum, is an oral endocannabinoid drug currently in Phase III trials for systemic sclerosis and dermatomyositis, among other indications.

Parion Sciences is a development-stage company focused on researching, developing, and commercializing treatments aimed at restoring the innate mucosal surface defenses of patients. These defenses are crucial for maintaining health in areas such as the eyes and lungs, where protective mucus plays a significant role. Parion's innovative technologies specifically target respiratory and ocular diseases that arise when the body's mucosal defense system is compromised due to factors like genetics, aging, or environmental influences. By addressing these unmet medical needs, Parion seeks to enhance the ability of healthcare professionals to effectively restore and protect patients' mucosal surfaces.

Corus Pharma is a biotech and specialty drug company that focuses on respiratory and infectious diseases. In other words, it is a development stage biopharmaceutical company that focuses on the development and commercialization of novel applications and formulations of known therapeutics to treat severe respiratory diseases. The products of the company are Corus 1020 and 1030. Corus 1020 is designed to treat respiratory infections in cystic fibrosis patients, using an inhalable form of an antibiotic called aztreonam. Corus 1030 is inhaled lidocaine for the treatment of patients suffering from the severe persistent forms of asthma. Corus Pharma is a U.S.-based company that was founded in 2001 and the company was acquired by Gilead Sciences on October 22, 2006.

Altus Pharmaceuticals is an information technology consulting firm that serves a diverse range of clients, including healthcare providers, manufacturing companies, and financial institutions. The firm positions itself as a strategic partner, providing expertise to help organizations navigate complex challenges and enhance their operational efficiency. With a focus on delivering impactful solutions, Altus aims to support its clients in achieving their goals while addressing the unique demands of their respective industries.