Boundless Bio is a company that develops novel cancer therapeutics intended to understand and treat untraceable cancers. It aims to be the biopharma company interrogating extrachromosomal DNA (ecDNA) biology to deliver transformative therapies to patients with previously intractable cancers.
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.
Be Biopharma pioneers engineered B cell medicines aimed at improving treatments for conditions like Hemophilia B and cancer. Their innovative approach utilizes precision genome editing to create durable and effective cell therapies.
CDR-Life is a Zurich-based biotech company specializing in the development of innovative therapeutic antibody fragments, with particular emphasis on immuno-oncology and ophthalmology. Operating from the Bio-Technopark in Schlieren-Zurich, the company focuses on creating tumor-selective immunotherapies. Its technology is designed to produce MHC-specific antibodies that target tumor-specific intracellular antigens, thereby enabling healthcare providers to deliver more precise and effective treatments for cancer patients.
Ansa Biotechnologies is manufactured via a chemical method that has remained essentially unchanged for 35 years and has approached a plateau. Ansa Biotechnologies is developing a novel DNA synthesis technology based on enzymes that will be more fast, accurate, and clean than existing methods. The technique promises to dramatically accelerate innovation in biological research and engineering.
Ocelot Bio brings new treatment options to patients with severe liver disease. It researches and discovers medicines that help in the treatment of hepatorenal syndrome, a complication of severe liver disease.
Plexium develops a targeted protein degradation platform to find new therapies for cancer and other diseases. Plexium's platform focuses on the discovery of next-generation TPD drugs that extend beyond the limited applications of Proteolysis Targeting Chimeras (PROTACs) and cereblon imids, unraveling the boundless possibilities of true drug-like protein degraders. From molecular glues to monovalent degraders, the possibilities are endless across a wide variety of therapeutic areas.
Qvella Corporation is a molecular diagnostics company based in Toronto, Canada, focused on revolutionizing clinical microbiology through innovative technologies. Founded in 2009 by a group of scientists and engineers, the company aims to significantly reduce the time required to obtain results for microbiological tests. Qvella's flagship technology, known as Field Activated Sample Treatment (FAST™), employs a unique electrical lysing and sample treatment method called e-lysis™. This approach allows for the rapid and direct detection of infectious agents from unenriched biological samples, such as whole blood, without the need for extensive sample processing. By delivering actionable results in clinically relevant time frames, Qvella's technology seeks to enhance patient outcomes and improve the overall practice of medicine.
Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.
Element Biosciences is a life science company that focuses on developing innovative genetic analysis tools. The company is developing a modular and high-performing DNA sequencing platform that will deliver high-quality data, workflow flexibility, and make next-generation sequencing technology more accessible. The company's proprietary approach to improving the signal-to-noise ratio allows us to provide groundbreaking innovations in surface chemistry, instrumentation, and biochemistry to drastically decrease the run and capital costs while delivering high sequencing data quality.
Born from the founders’ recognition that their diverse but complementary skills and experiences would enable them to successfully address challenges that others had not, the company set about solving a vexing problem: how to bring true proteomics to the world in a way that accelerates therapeutic development, dramatically improves medical diagnostics, and makes personalized and predictive medicine a reality. The extraordinary team at Nautilus represents a wide spectrum of disciplines and expertise, including protein chemists, chip designers, molecular biologists, data scientists, material scientists, biophysicists, optical engineers, microfluidics engineers, bioinformaticists, software engineers, and more. Nautilus is positioned to revolutionize proteomics, transform the way drugs are developed, and significantly improve the way human health is managed.
Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.
Boundless Bio is a company that develops novel cancer therapeutics intended to understand and treat untraceable cancers. It aims to be the biopharma company interrogating extrachromosomal DNA (ecDNA) biology to deliver transformative therapies to patients with previously intractable cancers.
New Equilibrium Biosciences provides a platform that reveals opportunities for structure-based design by learning biophysics with AI.
Scribe Therapeutics is a biotech company that develops and engineers new therapeutics based on CRISPR. The company’s first technology, called X-Editing (XE) molecules, are highly engineered CRISPR enzymes that provide combined aspects of greater efficacy, specificity, and deliverability than currently available CRISPR genome editing technology. It also a long-term, engineering approach that can help establish CRISPR-based therapies as a new standard of clinical care and expand access to groundbreaking treatments that will impact millions of lives. Scribe Therapeutics is overcoming the limitations of current genome editing technologies by developing custom-engineered enzymes and delivery modalities as part of a proprietary, evergreen platform for CRISPR-based genetic medicine. The company built for exceptional specificity within the genome – enabling therapeutic precision and allele-specific targeting. Scribe Therapeutics was founded in 2018 and is headquartered in Alameda, CA, USA.
Century Therapeutics builds an allogeneic iPSC-derived cell therapy platform that integrates gene editing, protein engineering, technical development, and manufacturing capabilities to generate allogeneic, iPSC-derived NK and T cell therapies for both hematological and solid tumor malignancies with significant unmet medical needs.
Vividion is a biotechnology company focused on developing innovative therapeutics that treat major unmet clinical needs using the first platform for proteome-wide drug and target discovery. Making accessible the broad set of proteins expressed in human cells, the company’s platform was spun out of the labs of Vividion’s scientific founders, a team of experts in chemical biology and synthetic chemistry from The Scripps Research Institute in La Jolla, CA. Vividion is committed to advancing and applying its pioneering synthetic and proteomic chemistry platforms to create therapeutics that will make a transformative difference in patients.
Day One Biopharmaceuticals is a biotechnology company that focuses on developing new cancer therapies for patients of all ages. The company understands that advances in cancer research now enable drug developers to identify children and adults who may benefit from the same medicine. They develop new therapies that meet the critical needs of people of all ages living with cancer starting from the biology of childhood cancer. Day One Biopharmaceuticals also focuses its efforts to bring the best medicines to families receiving the life-altering news of a new cancer diagnosis and the need for a treatment plan. It prioritizes programs that can be rapidly brought to market for both younger and older patients. The company's goal is to license and acquire the most promising products from research institutions as well as for biotechnology and pharmaceutical companies.
Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.
LianBio’s mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and major Asian markets through partnerships that provide access to the best science-driven therapeutic discoveries. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a pipeline based on disease relevance and the ability to impact patients with transformative mechanisms and precision-based therapeutics.
Be Biopharma pioneers engineered B cell medicines aimed at improving treatments for conditions like Hemophilia B and cancer. Their innovative approach utilizes precision genome editing to create durable and effective cell therapies.
Silverback Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, that specializes in developing tissue-targeted therapeutics aimed at treating cancer, chronic viral infections, and other serious diseases. The company’s lead product candidate, SBT6050, is currently undergoing a Phase I/Ib clinical trial; it is a TLR8 agonist conjugated to a HER2-directed monoclonal antibody, targeting tumors such as breast, gastric, and non-small cell lung cancers. Additionally, Silverback is advancing SBT6290, which is in preclinical development and targets Nectin4, found in bladder, triple-negative breast, head and neck, and non-small cell lung cancers. The company is also developing SBT8230, aimed at treating chronic hepatitis B virus infection, and exploring other agents that utilize its proprietary technology to modulate pathways in oncology and fibrosis indications. Silverback's innovative ImmunoTAC platform enables the design of therapies that are systemically delivered but act specifically at disease sites, unlocking previously inaccessible pathways for treatment.
Qvella Corporation is a molecular diagnostics company based in Toronto, Canada, focused on revolutionizing clinical microbiology through innovative technologies. Founded in 2009 by a group of scientists and engineers, the company aims to significantly reduce the time required to obtain results for microbiological tests. Qvella's flagship technology, known as Field Activated Sample Treatment (FAST™), employs a unique electrical lysing and sample treatment method called e-lysis™. This approach allows for the rapid and direct detection of infectious agents from unenriched biological samples, such as whole blood, without the need for extensive sample processing. By delivering actionable results in clinically relevant time frames, Qvella's technology seeks to enhance patient outcomes and improve the overall practice of medicine.
Saniona is a pharmaceutical company focusing on the development and advancement of multiple potential new medicines through clinical trials. The company is pioneering in the development of highly selective ion channel modulators. Ion channel modulation is a well-established approach to disease treatment that has been used in medications for diabetes, hypertension, epilepsy, and other conditions. TesometTM, the company's most advanced product candidate, has advanced to mid-stage clinical trials for rare eating disorders. Saniona is advancing two product candidates, SAN711 and SAN903, using its ion channel expertise. A Phase 1 clinical trial of SAN711 for the treatment of neuropathic pain conditions was completed successfully. SAN903 is now ready for Phase 1 clinical trials to treat inflammatory and fibrotic disorders.
Vor Biopharma, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing engineered hematopoietic stem cell (eHSC) therapies for cancer treatment. The company's lead product candidate, VOR33, is designed specifically for the treatment of acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, allowing for selective targeting of the cancer without harming normal cells. This innovative approach seeks to minimize the toxicities associated with traditional immunotherapies, which often affect both cancerous and healthy tissues. By protecting healthy stem cells from depletion while generating functional immune cells, Vor Biopharma aims to enhance therapeutic outcomes for patients with hematological malignancies.
Cybin is a clinical-stage biopharmaceutical company on a mission to create safe and effective psychedelic-based therapeutics to address the large unmet need for new and innovative treatment options for people who suffer from mental health conditions. Cybin’s goal of revolutionizing mental healthcare is supported by a network of world-class partners and internationally recognized scientists aimed at progressing proprietary drug discovery platforms, innovative drug delivery systems, and novel formulation approaches and treatment regimens. The Company is currently developing CYB003, a proprietary deuterated psilocybin analog for the treatment of major depressive disorder, and CYB004, a proprietary deuterated DMT molecule for generalized anxiety disorder, and has a research pipeline of investigational psychedelic-based compounds. Headquartered in Canada and founded in 2019, Cybin is operational in Canada, the United States, the United Kingdom, the Netherlands, and Ireland. For company updates and to learn more about Cybin, visit www.cybin.com or follow the team on Twitter, LinkedIn, YouTube, and Instagram.
Element Biosciences is a life science company that focuses on developing innovative genetic analysis tools. The company is developing a modular and high-performing DNA sequencing platform that will deliver high-quality data, workflow flexibility, and make next-generation sequencing technology more accessible. The company's proprietary approach to improving the signal-to-noise ratio allows us to provide groundbreaking innovations in surface chemistry, instrumentation, and biochemistry to drastically decrease the run and capital costs while delivering high sequencing data quality.
Legend Biotech is a clinical-stage biopharmaceutical company that discovers and develops novel cell therapies for oncology. The company develops advanced cell therapies across a diverse array of technology platforms, including autologous and allogeneic chimeric antigen receptor T-cell and natural killer (NK) cell-based immunotherapy. Its initial product candidate, LCAR-B38M/JNJ-4528, is a chimeric antigen receptor, or CAR, T cell therapy for the treatment of multiple myeloma.
Qvella Corporation is a molecular diagnostics company based in Toronto, Canada, focused on revolutionizing clinical microbiology through innovative technologies. Founded in 2009 by a group of scientists and engineers, the company aims to significantly reduce the time required to obtain results for microbiological tests. Qvella's flagship technology, known as Field Activated Sample Treatment (FAST™), employs a unique electrical lysing and sample treatment method called e-lysis™. This approach allows for the rapid and direct detection of infectious agents from unenriched biological samples, such as whole blood, without the need for extensive sample processing. By delivering actionable results in clinically relevant time frames, Qvella's technology seeks to enhance patient outcomes and improve the overall practice of medicine.
Vor Biopharma, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing engineered hematopoietic stem cell (eHSC) therapies for cancer treatment. The company's lead product candidate, VOR33, is designed specifically for the treatment of acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, allowing for selective targeting of the cancer without harming normal cells. This innovative approach seeks to minimize the toxicities associated with traditional immunotherapies, which often affect both cancerous and healthy tissues. By protecting healthy stem cells from depletion while generating functional immune cells, Vor Biopharma aims to enhance therapeutic outcomes for patients with hematological malignancies.
SeLux Diagnostics is a developer of the next-generation phenotyping platform that provides personalized antimicrobial therapies to patients. The company is revolutionizing patient care by expediting the selection of personalized antimicrobial therapy for all infectious disease patients. This advancement will save lives, reduce hospital stays, and address the escalating antibiotic resistance epidemic by minimizing the overuse of broad-spectrum agents.
89bio is a clinical-stage biopharmaceutical company focused on rapidly advancing their lead product candidate, BIO89-100, through clinical development for the treatment of non-alcoholic steatohepatitis (NASH). BIO89-100 is a specifically engineered glycoPEGylated analog of fibroblast growth factor 21 (FGF21). It may be a differentiated FGF21 therapy, and they believe it has the potential to become a mainstay of therapy for NASH given its ability to address the key liver pathologies (steatosis and fibrosis) and underlying metabolic dysregulation in patients with NASH. They are also expanding the breadth of indications for BIO89-100 with an initial focus on severe hypertriglyceridemia (SHTG).
Precision BioSciences is a biotechnology company dedicated to improving lives through its next-generation gene editing technology, ARCUS. Precision BioSciences’ mission is to translate the world’s most powerful genome editing technology into greatly needed products throughout the life sciences. Precision’s proprietary ARCUS genome editing technology enables the production of highly specific nucleases that can insert, remove, and modify DNA at essentially any location in a complex genome.
CiVi Biopharma is a clinical stage research and development biotechnology company whose mission is to create novel cardiovascular and metabolic therapies that have meaningful value to patients. The company has multiple assets in various stages of development including Intravenous Iloprost in Phase 3 trials for treatment of Systemic Sclerosis.
Synthorx, Inc. is a biopharmaceutical company based in La Jolla, California, focused on developing innovative cytokine Synthorin programs for the treatment of cancer and autoimmune disorders. The company's lead product candidate, THOR-707, is a variant of recombinant human IL-2 being explored for various solid tumors, both as a standalone treatment and in combination with immune checkpoint inhibitors. Additionally, Synthorx is working on IL-2 Synthorin for autoimmune conditions, IL-10 Synthorin for immuno-oncology, and IL-15 Synthorins to enhance immune regulation. The company employs advanced synthetic biology techniques, utilizing a novel DNA base pair to create proteins with enhanced specificity, safety, and efficacy. These developments enable the manufacturing of improved therapeutics that can better modulate pharmacological properties compared to traditional biologics. Synthorx, which was established in 2014 and previously known as Alinos, Inc., operates as a subsidiary of Sanofi and holds exclusive rights to its innovative synthetic biology technology derived from research at The Scripps Research Institute.
Qvella Corporation is a molecular diagnostics company based in Toronto, Canada, focused on revolutionizing clinical microbiology through innovative technologies. Founded in 2009 by a group of scientists and engineers, the company aims to significantly reduce the time required to obtain results for microbiological tests. Qvella's flagship technology, known as Field Activated Sample Treatment (FAST™), employs a unique electrical lysing and sample treatment method called e-lysis™. This approach allows for the rapid and direct detection of infectious agents from unenriched biological samples, such as whole blood, without the need for extensive sample processing. By delivering actionable results in clinically relevant time frames, Qvella's technology seeks to enhance patient outcomes and improve the overall practice of medicine.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.
Spero Therapeutics is developing first in class therapeutics for treatment of gram-negative infections. The company leverages a top-tier chemistry and microbiology team, an efficient virtual approach to drug development, and a focus on high potential, novel mechanisms to fill the unmet need for early stage therapeutics targeting serious bacterial infections. Its lead program addresses a novel target driving virulence and persistence of pseudomonas aeruginosa infections and other gram-negative pathogens.
Synthorx, Inc. is a biopharmaceutical company based in La Jolla, California, focused on developing innovative cytokine Synthorin programs for the treatment of cancer and autoimmune disorders. The company's lead product candidate, THOR-707, is a variant of recombinant human IL-2 being explored for various solid tumors, both as a standalone treatment and in combination with immune checkpoint inhibitors. Additionally, Synthorx is working on IL-2 Synthorin for autoimmune conditions, IL-10 Synthorin for immuno-oncology, and IL-15 Synthorins to enhance immune regulation. The company employs advanced synthetic biology techniques, utilizing a novel DNA base pair to create proteins with enhanced specificity, safety, and efficacy. These developments enable the manufacturing of improved therapeutics that can better modulate pharmacological properties compared to traditional biologics. Synthorx, which was established in 2014 and previously known as Alinos, Inc., operates as a subsidiary of Sanofi and holds exclusive rights to its innovative synthetic biology technology derived from research at The Scripps Research Institute.
Qvella Corporation is a molecular diagnostics company based in Toronto, Canada, focused on revolutionizing clinical microbiology through innovative technologies. Founded in 2009 by a group of scientists and engineers, the company aims to significantly reduce the time required to obtain results for microbiological tests. Qvella's flagship technology, known as Field Activated Sample Treatment (FAST™), employs a unique electrical lysing and sample treatment method called e-lysis™. This approach allows for the rapid and direct detection of infectious agents from unenriched biological samples, such as whole blood, without the need for extensive sample processing. By delivering actionable results in clinically relevant time frames, Qvella's technology seeks to enhance patient outcomes and improve the overall practice of medicine.
Wave Life Sciences (Nasdaq: WVE) is a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
Calimmune is a clinical-stage gene therapy company. The company’s lead therapeutic candidate, is a gene-based therapy engineered to control HIV infection and to protect individuals with HIV from progressing to AIDS. The therapy is currently being evaluated in Phase I/II studies. Cal-1 is designed to reduce production of CCR5, a protein on the surface of white blood cells that plays a critical role in enabling HIV to infect cells. It also has a second mechanism aimed at preventing viral fusion, the process by which the virus enters the cell. This dual approach was shown to be effective against broad strains of HIV in pre-clinical studies.
Precision BioSciences is a biotechnology company dedicated to improving lives through its next-generation gene editing technology, ARCUS. Precision BioSciences’ mission is to translate the world’s most powerful genome editing technology into greatly needed products throughout the life sciences. Precision’s proprietary ARCUS genome editing technology enables the production of highly specific nucleases that can insert, remove, and modify DNA at essentially any location in a complex genome.
Merus B.V., a biomedical company, engages in the discovery and development of antibody-based biopharmaceuticals. It develops Oligoclonics technology that offers a class of human antibodies called Oligoclonics. Oligoclonics is a concept whereby a mixture of various human therapeutic antibodies, directed to a common antigen are produced from a single cell clone.
Natera specializes in cell-free DNA testing and is driven by a passion for elevating the science and utility of prenatal testing. The advent of the Information Age has created a paradigm shift in the fields of computing and informatics. Surprisingly, medical diagnostics have been slow to integrate these advances into genetic testing.
Wave Life Sciences (Nasdaq: WVE) is a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
Ascendis Pharma has built a high value pipeline with the aim of changing clinical treatment paradigms. Using our innovative TransCon prodrug technology platform, we continue to identify new opportunities addressing areas of high unmet medical need. TransCon is applicable to all drug classes, and is currently being used to create superior prodrugs of proteins, peptides and small molecules. Depending on choice of TransCon Carrier, either systemic or localized drug exposure is achieved, while dosing frequency is determined by the choice of TransCon Linker. The prodrugs are new chemical entities with new patent life. Product development process is de-risked, as the prodrugs release the active component in its native and unmodified form, maintaining the original drug’s well known mode of action.
Sierra Oncology is a late-stage biopharmaceutical company on a mission to deliver targeted therapies that treat rare forms of cancer. The company harnesses its deep scientific expertise to identify compounds that target the root cause of disease. Sierra Oncology's team takes an evidence-based approach to understand the limitations of current treatments and explore new ways to change the cancer treatment paradigm. Sierra Oncology is transforming promise into patient impact.
Lumena Pharmaceuticals is developing oral therapeutics for rare liver diseases to improve liver function, relieve disease symptoms and dramatically impact patient health. The company’s clinical-stage product candidates carry a reduced risk of systemic toxicities by selectively targeting a transporter in the intestine and are designed to be minimally absorbed in the body. Lumena’s lead candidate, LUM001, has been extensively evaluated in other indications across 12 clinical studies in more than 1,400 subjects. Because of the extensive preclinical and clinical data package for a compound at this stage in development, LUM001 is positioned to rapidly progress through the clinic in pediatric and adult patients with several types of cholestatic liver disease. While Lumena’s primary focus is to develop novel treatments for patients with rare liver diseases, the company’s therapeutic approach also has promising potential in the treatment of metabolic diseases affecting the liver, a significant and growing health problem in children and adults.
Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.
AmpliPhi Biosciences is a biopharmaceutical company focused on the development of an internally generated pipeline of naturally occurring viruses called bacteriophage (phage) for the treatment of bacterial infection. Phage-based therapy provides a unique and proven approach to treating a wide variety of bacterial infections and in particular, drug-resistant strains of bacteria that are commonly found in the hospital setting. We have built a drug development and manufacturing platform designed to allow rapid development and production of multiple phage-based therapies. Our plan and success relies on our ability to identify and develop selected phage to eradicate targeted bacterial infections across a wide range of bacterial disease. Initially our phage discovery and development platform will focus efforts in acute and chronic lung, sinus and gastrointestinal (GI) infections. AmpliPhi maintains operations in Richmond, Virginia in the United States, in Colworth Science Park, near London in the United Kingdom, and outside Sydney, NSW, Australia.
Bluebird bio is a clinical-stage biotechnology company that develops gene therapies for severe genetic and rare diseases. The company's gene therapy procedures aim to genetically modify a patient's cells to fundamentally correct or address the genetic basis underlying a disease. Bluebird's revenue is derived from collaboration arrangements, research fees, license fees, and grant revenue. The company works with industry peers, strategic partners, and nonprofit organizations for the development and commercialization of its product candidates.
CellScape Corporation (formerly Saryna Medical Corp) is a privately funded company headquartered in Newark, California, in the heart of the biotech corridor of the San Francisco Bay Area. The Company has developed whole fetal cell technology to transform prenatal genetic testing using a simple blood test. Their vision is to make comprehensive prenatal genetic testing available to every woman without requiring an invasive procedure that might put the fetus at risk. Their leadership team is accomplished in bringing innovative, cost-effective medical and molecular diagnostics to market.
The company is applying innovative science to improve the quality of eggs (oocytes) and increase the success of in vitro fertilization (IVF). OvaScience's proprietary technology, exclusively licensed from Harvard Medical School and Massachusetts General Hospital, enables the use of a women's own cellular energy to improve egg quality. OvaScience was co-founded by Richard Aldrich, Michelle Dipp, M.D., Ph.D., and Christoph Westphal M.D., Ph.D., of Longwood Founders Fund, along with fertility expert and Harvard Medical School Professor Jonathan Tilly, Ph.D. of Massachusetts General Hospital, and aging expert and Harvard Medical School Professor David Sinclair, Ph.D. The company is backed by leading life science investors Longwood Founders Fund and Bessemer Venture Partners. The company has assembled a world-class team of leading reproductive endocrinologists, embryologists, scientists and advisers to bring this technology to patients.
Seres Therapeutics is a clinical-stage biotherapeutic company that develops ecobiotic therapeutic products. They engaged in developing biological drugs which are referred to as ecobiotic microbiome therapeutics. The company is a group of experts in microbiome research and drug development. Its executive team, board of directors, and scientific advisory board have a wealth of experience in the development, regulatory approval, and profitable commercialization of a wide range of therapeutic products.
T2 Biosystems is a private company developing a next-generation medical diagnostic platform that enables rapid and accurate diagnostic tests of all types on a single, easy-to-use instrument. The company's proprietary technology combines nanotechnology and miniaturized magnetic resonance (MR) technology and eliminates the need for costly and time-consuming sample preparation.
T2 Biosystems is a private company developing a next-generation medical diagnostic platform that enables rapid and accurate diagnostic tests of all types on a single, easy-to-use instrument. The company's proprietary technology combines nanotechnology and miniaturized magnetic resonance (MR) technology and eliminates the need for costly and time-consuming sample preparation.