Cystic Fibrosis Foundation

Owlstone Medical Ltd is a diagnostics company based in Cambridge, United Kingdom, specializing in non-invasive breath-based diagnostic tools aimed at the early detection of diseases, including cancer and respiratory conditions. The company develops a range of innovative products, including its Breath Biopsy platform, which utilizes advanced technology to identify volatile organic compounds in breath samples. Its offerings include FAIMS technology for medical applications, Lonestar for disease biomarker detection, and ReCIVA breath samplers for in vitro analysis. Owlstone Medical's technology has significant applications in various medical fields, such as lung cancer, bowel cancer, and gastrointestinal disorders. The company is also exploring the development of a portable hand-held device that promises high sensitivity and selectivity for chemical detection, with potential applications across multiple markets, including military and industrial sectors. Owlstone Medical aims to become a leader in non-invasive diagnostics, with ongoing collaborations and trials, including contracts with the UK National Health Service for lung cancer screening initiatives.

BiomX discovers and develops innovative microbiome therapeutics. The Company's mission is to develop novel therapeutics for preventing and treating diseases in which microbiome imbalances have been implicated. BiomX's microbiome modulation technologies are based on the cutting-edge innovative research of its scientific collaborators: Professor Rotem Sorek, PhD and Dr. Eran Elinav, MD, PhD both of the Weizmann Institute of Science and Professor Timothy K. Lu, MD, PhD of the Massachusetts Institute of Technology.

ReCode Therapeutics 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 employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

BiomX discovers and develops innovative microbiome therapeutics. The Company's mission is to develop novel therapeutics for preventing and treating diseases in which microbiome imbalances have been implicated. BiomX's microbiome modulation technologies are based on the cutting-edge innovative research of its scientific collaborators: Professor Rotem Sorek, PhD and Dr. Eran Elinav, MD, PhD both of the Weizmann Institute of Science and Professor Timothy K. Lu, MD, PhD of the Massachusetts Institute of Technology.

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 proprietary Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aiming to restore normal genetic function and address the underlying causes of various diseases. By targeting a broad spectrum of conditions with significant unmet medical needs, Prime Medicine seeks to revolutionize the field of genetic medicine through effective and efficient therapeutic solutions.

Clarametyx Biosciences, Inc. is a biotechnology company based in Columbus, Ohio, focused on developing targeted and immune-enabling biologic therapies to address persistent infections linked to biofilms. Founded in 2020, the company utilizes a novel technology platform designed to penetrate the protective layers surrounding bacteria, facilitating immune responses and enhancing the effectiveness of antibiotic interventions. Among its key developments is CMTX-001, a humanized monoclonal antibody currently in preclinical stages aimed at treating hospital-acquired pneumonia. Clarametyx's innovative approach seeks to combat life-threatening infections while addressing the growing concerns of antibiotic resistance.

ViaNautis is focused on creating innovative medications that can effectively cross biological barriers, particularly the blood-brain barrier (BBB). The company utilizes PolyNaut, a versatile nano-engineered polymer technology, to enhance intracellular delivery of therapeutics. Through the development of nanoparticle technology, ViaNautis encapsulates various therapeutic modalities within polymer nanovesicles, allowing for selective targeting of different tissues and cell types. This approach significantly improves the therapeutic efficacy of the delivered molecules, enabling clients to achieve better treatment outcomes. With partners like SomaServe, ViaNautis is paving the way for advanced medications that offer substantial therapeutic benefits by overcoming various biological barriers.

Arcturus Therapeutics is an RNA medicines company based in San Diego, California, founded in 2013. It specializes in the discovery and development of novel RNA therapeutics aimed at treating rare diseases, particularly in the areas of liver and respiratory care. The company leverages its proprietary Self-Transcribing and Replicating RNA (STARR) technology and Unlocked Nucleic Acid (UNA) chemistry to create RNA drug substances and nanoparticle-formulated products. Arcturus's pipeline includes several preclinical and clinical programs, such as LUNAR-OTC for ornithine transcarbamylase deficiency, LUNAR-CF for cystic fibrosis, and LUNAR-COV19, a vaccine candidate targeting the Coronavirus outbreak. The company holds a robust patent portfolio, encompassing 182 patents and applications globally, enabling it to target any gene in the human genome. Furthermore, Arcturus has established collaborations with various pharmaceutical partners to advance its therapeutic candidates across multiple disease indications.

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 combines advanced high-throughput experimental and computational methods to create tailored delivery vehicles. These vehicles are designed to effectively transport a wide range of genetic materials with specific targeting capabilities, facilitating research in functional chemistry and enhancing the design of therapeutically relevant gene delivery systems.

BiomX discovers and develops innovative microbiome therapeutics. The Company's mission is to develop novel therapeutics for preventing and treating diseases in which microbiome imbalances have been implicated. BiomX's microbiome modulation technologies are based on the cutting-edge innovative research of its scientific collaborators: Professor Rotem Sorek, PhD and Dr. Eran Elinav, MD, PhD both of the Weizmann Institute of Science and Professor Timothy K. Lu, MD, PhD of the Massachusetts Institute of Technology.

ReCode Therapeutics 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 employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

Gensaic is a redosable genomic medication company that seeks to reimagine gene therapy.

Aridis Pharmaceuticals, Inc. is a late-stage biopharmaceutical company based in San Jose, California, that specializes in the discovery and development of targeted immunotherapy using fully human monoclonal antibodies (mAbs) to address life-threatening infections. The company’s lead product candidate, AR-301, is currently undergoing Phase III trials for treating lung infections caused by S. aureus alphatoxin. Additionally, Aridis is advancing several other candidates, including AR-105, a fully human IgG1 mAb in Phase II trials targeting the gram-negative bacterium P. aeruginosa, and AR-101, which is also in Phase II trials for hospital-acquired pneumonia caused by the same pathogen. Other projects in development include AR-401, AR-201, and AR-501, which target various serious infections. Aridis employs proprietary technology platforms to identify and manufacture potent mAbs derived from patients who have successfully overcome infections, enabling the production of therapeutics that are optimized for efficacy without requiring genetic modification. Founded in 2003, Aridis Pharmaceuticals is committed to providing innovative solutions for critical infections.

Pulmocide Ltd, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.

Carmine Therapeutics is focused on developing a novel gene therapy platform that harnesses extracellular vesicles produced from red blood cells. This innovative approach aims to address limitations associated with traditional viral-based gene therapies, such as immunogenicity, small transgene capacity, and manufacturing challenges. The company's technology is designed to generate a diverse pipeline of gene therapies targeting hematology, oncology, and immunology, offering new treatment options for patients facing these medical conditions. By leveraging this proprietary platform, Carmine Therapeutics seeks to advance the development of a new class of medicines.

Destiny Pharma is a clinical-stage biotechnology company based in Brighton, United Kingdom, specializing in the discovery and development of innovative antimicrobial medicines aimed at treating infectious diseases. The company is advancing several drug candidates, including XF-73 Nasal, currently in Phase II trials for preventing post-surgical staphylococcal infections, and XF-73 Dermal, which is in Phase I trials for treating skin infections caused by antibiotic-resistant bacteria. Additionally, Destiny Pharma is developing XF-70 for conditions such as ventilator-associated pneumonia and cystic fibrosis, along with DPD-207 for bacterial and fungal eye infections. The company collaborates with various academic institutions and organizations, including the University of Southampton and China Medical System Holdings Limited, to explore new treatments for infections associated with antibiotic resistance. Destiny Pharma's focus on unique mechanisms of action for its XF series presents potential solutions in addressing the growing global healthcare challenge of drug-resistant bacteria.

Carbon Biosciences focuses on developing genetic medicines to address serious diseases. The company utilizes a proprietary platform that employs novel parvoviruses, which are designed to target specific tissues and carry larger therapeutic cargo. This approach minimizes the risk of neutralizing immunity and allows for repeat dosing, making it particularly effective for conditions such as cystic fibrosis, where targeting lung and upper airway tissues is crucial. By leveraging industrial-scale viral production platforms, Carbon Biosciences aims to extend the reach of gene therapies, offering innovative solutions for researchers and patients alike.

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 based in Israel is a Pontifax portfolio company established in 2013 by Dr. Silvia Noiman, that serves as its Executive Chairman and Dr. Shmuel Tuvia. The company, is a biopharmaceutical company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations including: Cystic fibrosis, Duchene Muscular Dystrophy, Usher syndrome, Ataxia-telangiectasia, Beta thalassemia, Tay-Sachs Hurler syndrome and many others. Eloxx Pharmaceuticals approach and knowhow allows moving rapidly from target drugs discovery program to clinical drug candidates. Building a robust pipeline of molecules designed for an array of nonsense genetic diseases. Translation of mRNA to a protein is the event where the cellular machinery, i.e. ribosome, ultimately dictates the quantity and timing for each protein to be produced. Thus, molecules that induce ribosomal readthrough overcome the nonsense mutations and allow production of a full-length functional protein. These molecules hold great therapeutic potential for the treatment of many genetic diseases.

Felix Biotechnology is working to accelerate the deployment of novel biotherapeutics to manage infectious diseases. Felix Biotechnology's bio-therapeutics targets antibiotic-resistant bacteria to manage infectious diseases precisely and effectively, allowing healthcare professionals to manage urgent microbial challenges in human health.

Feldan Therapeutics is a biopharmaceutical company focused on developing innovative treatments using its proprietary peptide-based technology platform, known as the Feldan Shuttle. This platform facilitates fast and safe intracellular delivery, allowing access to cellular components that are typically unreachable by conventional drugs. By leveraging the unique characteristics of the Feldan Shuttle, the company aims to create a new generation of therapeutic applications, as evidenced by its ongoing pipeline of clinical programs. Feldan Therapeutics is committed to advancing the field of intracellular therapeutics to improve patient outcomes.

SalioGen Therapeutics is a biotechnology company focused on developing gene therapies for inherited disorders. Utilizing its proprietary Exact DNA Integration Technology (EDIT), the company employs mammal-derived enzymes, known as Saliogase, to facilitate genome engineering. This innovative approach enables the delivery of any size gene through non-viral methods, offering significant advantages in manufacturing and application. SalioGen's technology aims to provide durable, safe, and affordable treatments, potentially transforming the landscape of care for patients with genetic conditions and enhancing the efficiency of cell therapy and biologics manufacturing in the healthcare sector.

Peptilogics, Inc. is a biotechnology company based in West Mifflin, Pennsylvania, that focuses on developing novel peptide therapeutics to combat multidrug-resistant bacterial infections. Established in 2013, Peptilogics employs a unique computational platform that facilitates drug discovery by exploring new chemical design spaces and ensuring the biological viability and manufacturability of potential therapeutics. The company's lead candidate, PLG0206, is a broad-spectrum anti-infective peptide that has received FDA Orphan Drug Designation and Qualified Infectious Disease Product Designation, targeting prosthetic joint infections, which represent a significant unmet medical need. Peptilogics aims to provide effective solutions to address the challenges posed by resistant bacteria and biofilms, ultimately improving treatment outcomes for patients.

BiomX discovers and develops innovative microbiome therapeutics. The Company's mission is to develop novel therapeutics for preventing and treating diseases in which microbiome imbalances have been implicated. BiomX's microbiome modulation technologies are based on the cutting-edge innovative research of its scientific collaborators: Professor Rotem Sorek, PhD and Dr. Eran Elinav, MD, PhD both of the Weizmann Institute of Science and Professor Timothy K. Lu, MD, PhD of the Massachusetts Institute of Technology.

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.

Pulmocide Ltd, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.

Armata Pharmaceuticals is a clinical-stage biotechnology company based in Marina del Rey, California, specializing in the development of targeted bacteriophage therapeutics to combat drug-resistant bacterial infections. Founded in 2005, the company addresses the growing threat of multidrug-resistant bacteria, which has been exacerbated by the overuse of antibiotics. This situation risks ushering in a post-antibiotic era where even common infections can become severe or life-threatening. Armata utilizes its proprietary bacteriophage technology to create pathogen-specific therapies, advancing a diverse pipeline of natural and synthetic phage candidates aimed at tackling infections caused by organisms such as Pseudomonas aeruginosa and Staphylococcus aureus. In partnership with Merck, Armata is also working on proprietary synthetic phage candidates targeting an undisclosed infectious disease agent.

Eloxx Pharmaceuticals based in Israel is a Pontifax portfolio company established in 2013 by Dr. Silvia Noiman, that serves as its Executive Chairman and Dr. Shmuel Tuvia. The company, is a biopharmaceutical company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations including: Cystic fibrosis, Duchene Muscular Dystrophy, Usher syndrome, Ataxia-telangiectasia, Beta thalassemia, Tay-Sachs Hurler syndrome and many others. Eloxx Pharmaceuticals approach and knowhow allows moving rapidly from target drugs discovery program to clinical drug candidates. Building a robust pipeline of molecules designed for an array of nonsense genetic diseases. Translation of mRNA to a protein is the event where the cellular machinery, i.e. ribosome, ultimately dictates the quantity and timing for each protein to be produced. Thus, molecules that induce ribosomal readthrough overcome the nonsense mutations and allow production of a full-length functional protein. These molecules hold great therapeutic potential for the treatment of many genetic diseases.

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 focused on developing therapeutics for resistant and difficult-to-treat infections. Founded in 1982 and headquartered in Bozeman, Montana, with an additional office in Vancouver, Canada, the company specializes in bismuth-thiols, which are designed to prevent microbial biofilms from forming on implanted medical devices and to manage biofilms in various infectious diseases. Microbion's product pipeline includes treatments for chronic and acute wound infections, particularly those related to diabetic foot ulcers, orthopedic implant-related infections, and other chronic wounds. The company also addresses the challenges posed by antibiotic-resistant microbes, including methicillin-resistant staphylococcus aureus, and aims to reduce infections associated with military wounds.

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 based in Israel is a Pontifax portfolio company established in 2013 by Dr. Silvia Noiman, that serves as its Executive Chairman and Dr. Shmuel Tuvia. The company, is a biopharmaceutical company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations including: Cystic fibrosis, Duchene Muscular Dystrophy, Usher syndrome, Ataxia-telangiectasia, Beta thalassemia, Tay-Sachs Hurler syndrome and many others. Eloxx Pharmaceuticals approach and knowhow allows moving rapidly from target drugs discovery program to clinical drug candidates. Building a robust pipeline of molecules designed for an array of nonsense genetic diseases. Translation of mRNA to a protein is the event where the cellular machinery, i.e. ribosome, ultimately dictates the quantity and timing for each protein to be produced. Thus, molecules that induce ribosomal readthrough overcome the nonsense mutations and allow production of a full-length functional protein. These molecules hold great therapeutic potential for the treatment of many genetic diseases.

Spexis AG is a clinical-stage biopharmaceutical company based in Switzerland that specializes in the discovery and development of antibiotics and other specialized pharmaceutical products aimed at severe or life-threatening diseases. Founded in 1996, the company is focused on addressing high unmet medical needs, particularly in the realm of antibiotic resistance and severe respiratory conditions. Spexis's lead product, murepavadin, is currently in Phase III development, targeting Pseudomonas aeruginosa, which has been classified as a critical priority pathogen by the World Health Organization. The company's operations are centered on research, development, and the prospective commercialization of respiratory therapeutics.

Calithera Biosciences is a clinical-stage biopharmaceutical company based in South San Francisco, California, dedicated to discovering and developing small molecule drugs targeting tumor metabolism and immunology for cancer treatment. The company's lead candidate, CB-839, is a glutaminase inhibitor currently in Phase II clinical trials aimed at treating solid tumors. Additionally, Calithera is advancing INCB001158, an oral arginase inhibitor in Phase I/II trials for hematology and oncology. Other notable projects include CB-280, intended for cystic fibrosis treatment, and CB-708, an inhibitor of CD73. The company has established collaborations, including a license agreement with Mars, Inc. for the development of arginase inhibitors and a clinical trial partnership with Bristol-Myers Squibb to evaluate nivolumab in combination with CB-839. Founded in 2010, Calithera is focused on building a pipeline of innovative cancer therapeutics that address critical pathways necessary for tumor growth and survival.

enGene, Inc. is a biotechnology company based in Vancouver, Canada, specializing in mucosal immunotherapy to address inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables the localized delivery of immune-modulating proteins to mucosal tissues, targeting conditions affecting the gastrointestinal tract, lung, and bladder. This innovative platform allows for the systemic release of proteins from the gut, which can be beneficial for treating a variety of immune disorders, as well as other conditions such as diabetes, anemia, and hemophilia. Founded in 1999, enGene has formed a strategic alliance with Takeda Pharmaceutical Company to further advance its therapeutic offerings.

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 is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.

Microbion Corporation is a biopharmaceutical company focused on developing therapeutics for resistant and difficult-to-treat infections. Founded in 1982 and headquartered in Bozeman, Montana, with an additional office in Vancouver, Canada, the company specializes in bismuth-thiols, which are designed to prevent microbial biofilms from forming on implanted medical devices and to manage biofilms in various infectious diseases. Microbion's product pipeline includes treatments for chronic and acute wound infections, particularly those related to diabetic foot ulcers, orthopedic implant-related infections, and other chronic wounds. The company also addresses the challenges posed by antibiotic-resistant microbes, including methicillin-resistant staphylococcus aureus, and aims to reduce infections associated with military wounds.

Crestone is a clinical-stage biopharmaceutical company focused on developing novel small molecule drugs for serious bacterial infections. Its research pipeline includes treatments for Clostridium difficile, antibiotic-resistant Gram-positive infections, and tuberculosis. The company aims to address significant unmet medical needs by utilizing innovative mechanisms in its drug candidates, which are designed to disrupt the cycle of bacterial growth and resistance. Through its efforts, Crestone seeks to enhance treatment outcomes for patients dealing with these challenging infections.

Laurent Pharmaceuticals Inc. is a private biopharmaceutical company based in Montreal, Canada, focused on developing orally-active drug candidates aimed at modulating the immuno-inflammatory response in cystic fibrosis patients. The company's lead product, LAU-7b, is a novel once-a-day oral formulation of fenretinide, designed to address the compromised immune-inflammatory response associated with cystic fibrosis, a condition that can lead to significant pulmonary insufficiency. Founded in 2012, Laurent Pharmaceuticals is in the clinical stage of drug development, working to provide innovative treatment options for this complex disease.

Enterprise Therapeutics Ltd. is a drug discovery company based in Brighton, United Kingdom, founded in 2014. It focuses on the research and development of innovative therapies for respiratory diseases, specifically targeting chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. The company aims to create disease-modifying therapies that address the underlying mechanisms of mucus congestion in the lungs, which can lead to breathing difficulties and increased susceptibility to lung infections. By developing these novel treatments, Enterprise Therapeutics seeks to enhance the quality of life for patients suffering from these chronic respiratory conditions.

Eloxx Pharmaceuticals based in Israel is a Pontifax portfolio company established in 2013 by Dr. Silvia Noiman, that serves as its Executive Chairman and Dr. Shmuel Tuvia. The company, is a biopharmaceutical company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations including: Cystic fibrosis, Duchene Muscular Dystrophy, Usher syndrome, Ataxia-telangiectasia, Beta thalassemia, Tay-Sachs Hurler syndrome and many others. Eloxx Pharmaceuticals approach and knowhow allows moving rapidly from target drugs discovery program to clinical drug candidates. Building a robust pipeline of molecules designed for an array of nonsense genetic diseases. Translation of mRNA to a protein is the event where the cellular machinery, i.e. ribosome, ultimately dictates the quantity and timing for each protein to be produced. Thus, molecules that induce ribosomal readthrough overcome the nonsense mutations and allow production of a full-length functional protein. These molecules hold great therapeutic potential for the treatment of many genetic diseases.

Celtaxsys, Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapeutics for inflammatory diseases, cancer, and autoimmune disorders. Established in 2007 and headquartered in Atlanta, Georgia, the company is engaged in developing drug compounds that manipulate immune cell migration and activation. One of its key products, CTX-4430, is a small-molecule drug designed to inhibit the enzyme Leukotriene A4 hydrolase in immune cells, targeting conditions such as cystic fibrosis and moderately severe acne. Celtaxsys aims to provide innovative treatments that address the protective mechanisms of certain tumors, thereby enhancing the immune response against cancer and managing serious inflammatory conditions.

Spirovant is focused on changing the course of cystic fibrosis and other pulmonary diseases. Their gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis.

Vast Therapeutics develops inhalable treatments for antibiotic-resistant pathogens present in cystic fibrosis and other respiratory disease patients. The company is in the preclinical stage of developing inhalable therapeutics to treat chronic infections common to the cystic fibrosis population.

Aridis Pharmaceuticals, Inc. is a late-stage biopharmaceutical company based in San Jose, California, that specializes in the discovery and development of targeted immunotherapy using fully human monoclonal antibodies (mAbs) to address life-threatening infections. The company’s lead product candidate, AR-301, is currently undergoing Phase III trials for treating lung infections caused by S. aureus alphatoxin. Additionally, Aridis is advancing several other candidates, including AR-105, a fully human IgG1 mAb in Phase II trials targeting the gram-negative bacterium P. aeruginosa, and AR-101, which is also in Phase II trials for hospital-acquired pneumonia caused by the same pathogen. Other projects in development include AR-401, AR-201, and AR-501, which target various serious infections. Aridis employs proprietary technology platforms to identify and manufacture potent mAbs derived from patients who have successfully overcome infections, enabling the production of therapeutics that are optimized for efficacy without requiring genetic modification. Founded in 2003, Aridis Pharmaceuticals is committed to providing innovative solutions for critical infections.

Laurent Pharmaceuticals Inc. is a private biopharmaceutical company based in Montreal, Canada, focused on developing orally-active drug candidates aimed at modulating the immuno-inflammatory response in cystic fibrosis patients. The company's lead product, LAU-7b, is a novel once-a-day oral formulation of fenretinide, designed to address the compromised immune-inflammatory response associated with cystic fibrosis, a condition that can lead to significant pulmonary insufficiency. Founded in 2012, Laurent Pharmaceuticals is in the clinical stage of drug development, working to provide innovative treatment options for this complex disease.

Icagen, Inc. is a drug discovery company based in Durham, North Carolina, specializing in neuroscience and rare diseases. Founded in 2003, the company focuses on developing and commercializing novel orally-administered small molecule drugs that target ion channels. Icagen provides a range of pre-clinical drug discovery services, including assay development, cell line generation, high-throughput screening, and medicinal chemistry. The company is actively engaged in research collaborations and partnerships with pharmaceutical and biotechnology firms to advance the discovery and development of pharmaceuticals. Icagen is currently developing several drug candidates, including ICA-105665, which is in Phase I trials for epilepsy and neuropathic pain, and Senicapoc, undergoing Phase I studies for asthma. Their ongoing programs also target new therapeutics for pain and inflammatory disorders, supported by collaboration agreements with major pharmaceutical companies.

Laurent Pharmaceuticals Inc. is a private biopharmaceutical company based in Montreal, Canada, focused on developing orally-active drug candidates aimed at modulating the immuno-inflammatory response in cystic fibrosis patients. The company's lead product, LAU-7b, is a novel once-a-day oral formulation of fenretinide, designed to address the compromised immune-inflammatory response associated with cystic fibrosis, a condition that can lead to significant pulmonary insufficiency. Founded in 2012, Laurent Pharmaceuticals is in the clinical stage of drug development, working to provide innovative treatment options for this complex disease.

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 is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.

Sound Pharmaceuticals, Inc. is a biopharmaceutical company based in Seattle, Washington, that specializes in developing neurotologic drugs aimed at treating sensorineural diseases of the inner ear. Established in 2001, the company focuses on creating prescription medications that help prevent and treat hearing loss. Its innovative platform works by developing compounds that antagonize growth inhibitory proteins, allowing normally non-dividing epithelial cells in the inner ear to divide and potentially become replacement auditory hair cells. This process, which does not occur naturally in humans after birth, offers a new approach to addressing various forms of hearing loss in a cost-effective manner.

Aridis Pharmaceuticals, Inc. is a late-stage biopharmaceutical company based in San Jose, California, that specializes in the discovery and development of targeted immunotherapy using fully human monoclonal antibodies (mAbs) to address life-threatening infections. The company’s lead product candidate, AR-301, is currently undergoing Phase III trials for treating lung infections caused by S. aureus alphatoxin. Additionally, Aridis is advancing several other candidates, including AR-105, a fully human IgG1 mAb in Phase II trials targeting the gram-negative bacterium P. aeruginosa, and AR-101, which is also in Phase II trials for hospital-acquired pneumonia caused by the same pathogen. Other projects in development include AR-401, AR-201, and AR-501, which target various serious infections. Aridis employs proprietary technology platforms to identify and manufacture potent mAbs derived from patients who have successfully overcome infections, enabling the production of therapeutics that are optimized for efficacy without requiring genetic modification. Founded in 2003, Aridis Pharmaceuticals is committed to providing innovative solutions for critical infections.

Laurent Pharmaceuticals Inc. is a private biopharmaceutical company based in Montreal, Canada, focused on developing orally-active drug candidates aimed at modulating the immuno-inflammatory response in cystic fibrosis patients. The company's lead product, LAU-7b, is a novel once-a-day oral formulation of fenretinide, designed to address the compromised immune-inflammatory response associated with cystic fibrosis, a condition that can lead to significant pulmonary insufficiency. Founded in 2012, Laurent Pharmaceuticals is in the clinical stage of drug development, working to provide innovative treatment options for this complex disease.

Laurent Pharmaceuticals Inc. is a private biopharmaceutical company based in Montreal, Canada, focused on developing orally-active drug candidates aimed at modulating the immuno-inflammatory response in cystic fibrosis patients. The company's lead product, LAU-7b, is a novel once-a-day oral formulation of fenretinide, designed to address the compromised immune-inflammatory response associated with cystic fibrosis, a condition that can lead to significant pulmonary insufficiency. Founded in 2012, Laurent Pharmaceuticals is in the clinical stage of drug development, working to provide innovative treatment options for this complex disease.

Corbus Pharmaceuticals is a clinical-stage pharmaceutical company based in Norwood, Massachusetts, that focuses on developing and commercializing therapeutics for inflammatory and fibrotic diseases. Founded in 2009, the company’s lead product candidate, lenabasum, is a synthetic oral CB2 agonist currently undergoing Phase III clinical trials for systemic sclerosis and dermatomyositis, and Phase IIb trials for systemic lupus erythematosus and cystic fibrosis. In addition to lenabasum, Corbus is developing CRB-4001, a peripherally-restricted CB1 inverse agonist aimed at treating nonalcoholic steatohepatitis and other fibrotic conditions. The company is leveraging a diverse pipeline of drug candidates sourced from a library of over 600 novel compounds targeting the endocannabinoid system. Furthermore, Corbus has established collaborations for the development and commercialization of its products, including a partnership with Kaken Pharmaceutical Co., Ltd. for lenabasum in Japan.

Parion Sciences is a development stage company focused on researching, developing, and commercializing treatments aimed at restoring the innate mucosal surface defenses in patients. The company's innovative technologies specifically target diseases affecting the respiratory and ocular systems, where the protective mucus layer is compromised due to factors such as genetics, aging, or environmental influences. By addressing these unmet medical needs, Parion Sciences seeks to enhance the health and well-being of individuals suffering from conditions related to weakened mucosal defenses.

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.

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