Carbon Biosciences develops genetic medicines for the treatment of devastating diseases. They develop their vector using industrial-scale viral production platforms and can be used to deliver a wide-range of 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 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 has developed a patented peptide-based technology platform, the Feldan Shuttle, which enables fast and safe intracellular delivery. The company's mission is to develop leading-edge therapeutic applications based on its proprietary platform, as demonstrated by its current pipeline of clinical programs taking advantage of the unique characteristics of the Shuttle.
SalioGen Therapeutics is a gene coding company that develops potentially curative therapies for patients with inherited disorders. Its Gene Coding approach is guided by our Exact DNA Integration Technology (EDIT) platform, a genome engineering technology that leverages proprietary mammal-derived enzymes they call Saliogase. With broad applicability, non-viral delivery capabilities of any size gene, and significant manufacturing advantages, EDIT has the potential to transform the treatment paradigm for inherited disorders and beyond.
Peptilogics is a clinical-stage biotechnology company that designs and develops novel peptide therapeutics. Through their computational platform, Peptilogics is radically altering drug discovery by not only uncovering new, previously unexplored chemical design space, but ensuring that each potential therapeutic is biologically viable and scalable to manufacture. Peptilogics' lead clinical stage peptide therapeutic, PLG0206, is a novel, broad-spectrum anti-infective that has been granted FDA Orphan Drug Designation and Qualified Infectious Disease Product Designation for its initial focus on the treatment of prosthetic joint infections, an unmet medical need.
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 is a product-focused bioengineering company that advances front-line labeled products for chronic, recurrent, and life-threatening pulmonary infections. Through breakthrough systems and synthetic biology platforms, EnBiotix is transforming and revitalizing existing antibiotic classes to combat the global rise of drug-resistant bacterial infections. These approaches have developed a robust clinical product pipeline addressing a wide range of acute and chronic infections in what is today over a $40 billion market, with the potential to significantly impact the lives of patients, caregivers, and payors worldwide. It was founded in 2012 and is headquartered in Cambridge, Massachusetts.
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 focused on the development of precisely targeted bacteriophage therapeutics for the treatment of drug-resistant bacterial infections. Antibiotic overuse has led to the marked rise of multidrug-resistant bacteria, putting us on the verge of a post-antibiotic era in which common infections can be serious or life-threatening. Additionally, the use of broad-spectrum antibiotics is known for disrupting the ecology of the human microbiome, leading to disease. The company was founded in 2005 and is based in Marina del Rey, CA, USA.
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 uses metagenomics and machine learning to discover novel genome editing systems for therapeutics development. The company's metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. Metagenomi forges these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific, and have a decreased risk of the immune response. Metagenomi's goal is to revolutionize gene editing for the benefit of patients around the world. The company was founded in 2018 and is headquartered in Emeryville, California.
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 is a pharmaceutical company that focuses on the development of therapeutics for the treatment of bacterial infections. It develops bismuth-thiols to prevent microbial biofilms from forming on medical devices implanted in the body and to prevent microbial biofilms in agricultural crops and industrial surfaces, including ships’ hulls, pulp and paper, and oil industries. It was founded in 1982 and 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 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.
Polyphor is a Swiss Pharma company focusing on the discovery and development of macrocycle drugs addressing high unmet medical needs. Polyphor was founded in 1996 and is a clinical stage, privately held Swiss specialty pharma company, based in Allschwil near Basel. It focuses on the development of macrocycle drugs that address antibiotic resistance and severe respiratory diseases.
Calithera Biosciences is a development-stage pharmaceutical company committed to discovering and developing novel small molecule therapeutics for the treatment of cancer. They are applying Their scientific expertise to build a pipeline of unique anti- cancer drugs that selectively target metabolic and apoptotic pathways critical to tumor growth and survival.
enGene is a developer of a mucosal immunotherapy platform designed to treat inflammatory bowel disease and diabetes. The company's mucosal immunotherapy platform has developed a flexible nucleotide delivery technology targeting mucosal tissues to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels, enabling physicians and doctors to regenerate physiologic, meal-regulated insulin secretion from the gut of subjects with diabetes.
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 biotechnology company.The Company designs, develops, and commercializes transformative gene therapeutic products for unmet medical conditions.
Microbion is a pharmaceutical company that focuses on the development of therapeutics for the treatment of bacterial infections. It develops bismuth-thiols to prevent microbial biofilms from forming on medical devices implanted in the body and to prevent microbial biofilms in agricultural crops and industrial surfaces, including ships’ hulls, pulp and paper, and oil industries. It was founded in 1982 and headquartered in Bozeman, Montana.
Crestone is a clinical-stage biopharmaceutical business that is developing novel medication candidates. The following are in the company's research pipeline: C. Treatments for C. diff, antibiotic-resistant Gram-positive infections, and tuberculosis.
Laurent Pharmaceuticals is a private biopharmaceutical company developing a clinical-stage, orally-active drug candidate. The company has a clinical-stage drug development program (LAU-7b) that addresses the compromised immune-inflammatory response in Cystic Fibrosis (CF), a critical but still enigmatic pathogenic pathway leading to pulmonary insufficiency in CF patients. Laurent Pharmaceuticals was founded in 2012 and is headquartered in Montréal, Quebec.
Enterprise Therapeutics is a drug discovery company dedicated to the research and development of novel therapies for the treatment of respiratory diseases. In diseases such as cystic fibrosis, asthma and COPD the lungs become congested with mucus leading to difficulty in breathing. New disease modifying therapies that target the underlying mechanisms of mucus congestion will reduce the frequency of lung infections and improve patient quality of life.
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, is a biopharmaceutical company, engages in developing and testing drug compounds for treating cancer, and inflammatory and autoimmune diseases. Its technology manipulates immune cell migration as a treatment for cancer, inflammation, and organ/tissue rejection. The company's drug compounds are designed to block the protective mechanisms created by certain tumors to prevent attack by immune system cells.
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.
Laurent Pharmaceuticals is a private biopharmaceutical company developing a clinical-stage, orally-active drug candidate. The company has a clinical-stage drug development program (LAU-7b) that addresses the compromised immune-inflammatory response in Cystic Fibrosis (CF), a critical but still enigmatic pathogenic pathway leading to pulmonary insufficiency in CF patients. Laurent Pharmaceuticals was founded in 2012 and is headquartered in Montréal, Quebec.
Icagen, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of novel orally-administered small molecule drugs that modulate ion channel targets. It develops ICA-105665 that is in Phase I single dose escalation study in healthy volunteers for epilepsy and neuropathic pain; and Senicapoc, which is in Phase I multiple dose study in healthy volunteers for the treatment of asthma. The company also conducts ongoing drug discovery programs on new therapeutics for pain and inflammatory disorders. Icagen has collaboration agreements with Bristol-Myers Squibb Company; Astellas Pharma, Inc.; and Pfizer, Inc.
Laurent Pharmaceuticals is a private biopharmaceutical company developing a clinical-stage, orally-active drug candidate. The company has a clinical-stage drug development program (LAU-7b) that addresses the compromised immune-inflammatory response in Cystic Fibrosis (CF), a critical but still enigmatic pathogenic pathway leading to pulmonary insufficiency in CF patients. Laurent Pharmaceuticals was founded in 2012 and is headquartered in Montréal, Quebec.
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 Inc. is a biopharmaceutical company based in Austin, Texas, specializing in rare respiratory diseases. Its primary product candidate, Molgradex, is an inhaled granulocyte-macrophage colony-stimulating factor currently in Phase III development for autoimmune pulmonary alveolar proteinosis (aPAP). Additionally, Molgradex is being evaluated in Phase IIa trials for nontuberculous mycobacterial (NTM) lung infections and cystic fibrosis patients with chronic NTM infections. Savara is also developing AeroVanc, an inhaled vancomycin, which is in Phase III trials for treating methicillin-resistant Staphylococcus aureus (MRSA) lung infections in cystic fibrosis patients. The company aims to expand its pipeline through strategic partnerships and product acquisitions, including a recent acquisition of a combination antibiotic that targets drug-resistant pathogens. Savara's management team has extensive experience in orphan drug development and is focused on addressing unmet medical needs in the field of pulmonary medicine.
4D Molecular Therapeutics is a biotechnology company.The Company designs, develops, and commercializes transformative gene therapeutic products for unmet medical conditions.
Sound Pharmaceuticals is a biopharmaceutical company that develops prescription drugs that enable doctors and patients to prevent and treat hearing loss. It focuses on developing drug treatments for sensorineural hearing loss.
Laurent Pharmaceuticals is a private biopharmaceutical company developing a clinical-stage, orally-active drug candidate. The company has a clinical-stage drug development program (LAU-7b) that addresses the compromised immune-inflammatory response in Cystic Fibrosis (CF), a critical but still enigmatic pathogenic pathway leading to pulmonary insufficiency in CF patients. Laurent Pharmaceuticals was founded in 2012 and is headquartered in Montréal, Quebec.
Laurent Pharmaceuticals is a private biopharmaceutical company developing a clinical-stage, orally-active drug candidate. The company has a clinical-stage drug development program (LAU-7b) that addresses the compromised immune-inflammatory response in Cystic Fibrosis (CF), a critical but still enigmatic pathogenic pathway leading to pulmonary insufficiency in CF patients. Laurent Pharmaceuticals was founded in 2012 and is headquartered in Montréal, Quebec.
Corbus Pharmaceuticals Holdings, Inc. is a Phase 3 clinical-stage pharmaceutical company focused on the development and commercialization of novel therapeutics to treat inflammatory and fibrotic diseases by leveraging its pipeline of endocannabinoid system-targeting synthetic drug candidates. The Company's lead product candidate, lenabasum, is a novel, synthetic, oral, selective cannabinoid receptor type 2 (CB2) agonist designed to resolve chronic inflammation and fibrotic processes. Lenabasum is currently being evaluated in systemic sclerosis, cystic fibrosis, dermatomyositis, and systemic lupus erythematosus. Corbus is also developing a pipeline of drug candidates from more than 600 novel compounds targeting the endocannabinoid system. The pipeline includes CRB-4001, a 2nd generation, peripherally-restricted, selective cannabinoid receptor type 1 (CB1) inverse agonist. Potential indications for CRB-4001 include NASH, among others. Corbus plans to start a Phase 1 study of CRB-4001 in 2019, intended to be followed by a National Institutes of Health (NIH)-funded proof-of-concept Phase 2 study.
Parion Sciences is a development stage company dedicated to research, development, and commercialization of treatments to restore patient’s innate mucosal surface defenses. In the eye and in the lung, our layer of protective mucus plays an important role in keeping our bodies healthy. Sometimes though, either through genetics, aging or environmental stimulus, our innate mucosal defense system can become compromised. Parion’s science driven technologies target respiratory and ocular diseases in which the patient’s ability to protect their mucosal surfaces is compromised
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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