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.
Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on advancing new therapies for millions of patients living with inflammatory diseases and autoimmune disorders. Ventyx’s clinical stage pipeline includes VTX958, a Phase 1 allosteric TYK2 inhibitor for the treatment of a broad range of autoimmune diseases, VTX002, a Phase 2-ready S1P1 receptor modulator for the treatment of ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is a mediator of multiple inflammatory conditions.
Elektrofi is a preclinical-stage company dedicated to redefining the standard of drug delivery by transforming the patient experience. Elektrofi’s formulation system can effectively process a wide range of therapeutic molecules. Their technology has the capacity to process large, increasingly complex and delicate proteins including monoclonal antibodies, bispecifics and fusion proteins. Founded in 2016, Elektrofi is based in Boston, Massachusetts, United States.
Triana Biomedicines is a developer of a molecular glue discovery platform to regulate disease targets that are difficult to address with any other modality. Triana’s drug discovery engine is powered by high-resolution structural insights, state-of-the-art in silico tools, and bespoke chemical libraries. The research team has validated the best-in-class platform and initiated multiple programs across different disease areas.
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s full-stack platform vertically integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance both basic research and therapeutic development programs. By providing both commercial and academic researchers and therapeutic developers with unprecedented access to cutting-edge genome engineering, Synthego is at the forefront of innovation, accelerating the development of truly engineered biology. The company was founded in 2012 and is based in Redwood City, California.
Parse Bio is a biotechnology startup company developing single-cell sequencing kit solutions for researchers. By using combinatorial cDNA barcoding within cells themselves, Parse Biosciences’s technology allows researchers to eliminate the need for complex and expensive microfluidic instruments. Additionally, the company’s technology enables samples to be fixed, frozen, and stored, allowing researchers to separate out the sample extraction from downstream library preparation steps. This capability empowers researchers to run fixed samples collected on different dates together in a single experiment, providing greater control over sample handling and reducing handling errors. Split Biosciences rebranded as Parse Biosciences in 2020. The Seattle, Washington-based biotech company was established in 2018 by Alex Rosenberg, Charlie Roco, and Georg Friedrich Seelig.
Septerna is a biotechnology company that discovers and advances novel small-molecule medicines targeting G protein-coupled receptors. The company's mission is to discover, develop, and commercialize medicines that improve the lives of patients by combining GPCR drug targets with the power of state-of-the-art small molecule drug discovery technologies. Septerna’s Native Complex platform is the engine that will make this happen.
Simcha Therapeutics is a biopharmaceutical company developing engineered cytokine immunotherapy for cancer. The company uses directed evolution to engineer novel cytokines designed to unlock the precision and power of the immune system. Simcha’s lead program, ST-067, is a designer IL-18 cytokine that has shown potent antitumor effects in animal models, both as a monotherapy and when combined with anti-PD-1 checkpoint inhibitors, as described in Nature in June 2020. It was founded in 2018 and is headquartered in New Haven, Connecticut, USA.
Enliven Therapeutics is a precision oncology company developing small molecule therapies to extend and improve patient lives. The company's discovery process combines clinically validated biological targets and industry-leading chemistry to address the limitations of existing therapies. This approach allows us to efficiently and effectively develop precision oncology therapeutics that enhance overall patient well-being. Founded in July 2019 by Sam Kintz, MBA, Joe Lyssikatos, PhD, and Anish Patel, PharmD, Enliven Therapeutics is based in Boulder, Colorado.
Quanta is a biotechnology company uncovering novel cancer therapeutic candidates to target driver oncogenes. It develops allosteric modulation to directly restore conformational control of active RAS signaling at the cell membrane to inhibit the full scope of RAS-driven cancer types.
Cedilla is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation. The company was founded in 2018 and based in Cambridge, Massachusetts.
Neumora Therapeutics operates as a clinical-stage biotechnology company pioneering precision medicines for brain diseases through the integration of data science and neuroscience.
Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases.
858 Therapeutics is a biotechnology company focused on discovering small molecule therapeutics for novel targets in oncology and immunology. The company emphasizes innovative approaches in synthetic lethality, innate immunity, and RNA modulation. Its lead program is a PARG inhibitor currently in early clinical development.
Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on advancing new therapies for millions of patients living with inflammatory diseases and autoimmune disorders. Ventyx’s clinical stage pipeline includes VTX958, a Phase 1 allosteric TYK2 inhibitor for the treatment of a broad range of autoimmune diseases, VTX002, a Phase 2-ready S1P1 receptor modulator for the treatment of ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is a mediator of multiple inflammatory conditions.
Alpine Immune Sciences is a clinical-stage biopharmaceutical business focused on researching and developing protein-based immunotherapies to treat autoimmune and inflammatory disorders. Its strategy incorporates a patented scientific platform for transforming native immune system proteins into differentiated, multi-targeted therapies. ALPN-303, or povetacicept, is a dual antagonist of the B cell activating factor, or BAFF, and a proliferation-inducing ligand, or APRIL, cytokines, which play key roles in the activation, development, and survival of B cells, and ALPN-101, or acazicolcept, is a dual Inducible T cell Costimulator, or ICOS, and CD28 antagonist intended for the treatment of autoimmune and inflammatory diseases.
Vanqua Bio is a biotechnology company that discovers and develops next-generation medicines for patients with neurodegenerative diseases. The company's technology platform utilizes human genetics and patient-derived neuronal cells to identify, validate, and clinically translate novel disease pathways associated with lysosomal dysfunction or aberrant activation of the innate immune system.
RareCyte is a life science research and diagnostic development company focused on characterizing and isolating rare cells in the blood. RareCyte is dedicated to bringing its innovative technology to the Life Sciences research, drug development, and diagnostic markets providing new tools to answer difficult questions related to cancer and cardiovascular disease. RareCyte's AccuCyte system is an open platform for the unbiased discovery and isolation of circulating tumor cells (CTCs) and circulating endothelial cells (CECs) from whole blood.
Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.
Attralus is a biopharmaceutical company focusing on creating transformative medicines to improve the lives of patients with systemic amyloidosis. Its proprietary peptide-based pan-amyloid targeting agents have the potential to diagnose and treat all forms and stages of systemic amyloidosis. The company is focused on targeting common pathology in all systemic amyloidosis diseases, with the goal of developing treatments for all subtypes of amyloidosis, including the majority in which there are currently no treatment options.
Asher Bio is an information technology company that specializes in the fields of CFO Office transformation, profitability, and cost management. It focuses on building and developing better immunotherapy for cancer. The company was founded in 2019 and headquartered in San Francisco, California.
DICE Therapeutics is a biopharmaceutical company leveraging its proprietary technology platform to build a pipeline of novel oral therapeutic candidates to treat chronic diseases in immunology and other therapeutic areas. DICE is initially focused on developing oral therapeutics against well-validated targets in immunology, with the goal of achieving comparable potency to their systemic biologic counterparts, which have demonstrated the greatest therapeutic benefit to date in these disease areas. The Company’s DELSCAPE platform is designed to discover selective oral small molecules with the potential to modulate protein-protein interactions (PPIs) as effectively as systemic biologics. DICE’s lead therapeutic candidate, S011806, is an oral antagonist of the pro-inflammatory signaling molecule, interleukin-17 (IL-17), which is a validated drug target implicated in a variety of immunology indications. DICE is also developing oral therapeutic candidates targeting α4ß7 integrin and αVß1/αVß6 integrin for the treatment of inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.
Tango Therapeutics operator of a biotechnology company created to discover novel drug targets and deliver the next generation of targeted therapies to people with cancer. The company's research leverages the genetic principle of synthetic lethality to discover and develop therapies that take aim at driver genes in cancer that are focused on three core areas, including counteracting tumor suppressor gene loss, reversing the ability of cancer cells to evade the immune system and identifying novel combinations, enabling patients to get therapies that are more effective than single-agent therapy.
Zentera Therapeutics is a biopharmaceutical company.
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.
Chronicle is a next-generation NFT platform that prioritizes ease of use, allowing anyone to buy, sell, and trade collections from leading brands.
Interius BioTherapeutics is a biopharmaceutical company that develops in vivo cell-specific gene medicines to treat B cell lymphomas. Intravenous in vivo CAR treatment, which treats B cell lymphomas, is the company's flagship program. Its unique engineering provides exceptional target tissue specificity. The business is working on a second initiative to address autoimmune conditions. With the help of Interius, a novel and distinct therapeutic modality for the precise administration of gene therapies may soon be made available to patients in expanded care settings, without the need for preconditioning chemotherapy.
Nuvalent is a biotechnology company that develops targeted therapies for clinically proven kinase targets in cancer. Leveraging deep expertise in structure-based design, Nuvalent develops innovative small molecules with exquisite target selectivity to overcome resistance, minimize adverse events, and drive more durable responses. It is advancing a robust pipeline with parallel lead programs in ROS1-positive and ALK-positive NSCLC, along with multiple discovery-stage research programs.
Gennao Bio is a privately held genetic medicines company developing first-in-class targeted nucleic acid therapeutics utilizing a proprietary gene monoclonal antibody (GMAB) platform technology. GMAB is an adaptive technology that uses a novel, cell-penetrating antibody to non-covalently bind to and deliver therapeutic levels of a wide variety of nucleic acid payloads to select cells. This non-viral delivery platform is differentiated from traditional gene delivery systems as it can deliver multiple types of nucleic acids, allows for repeat dosing and employs well-established manufacturing processes. Gennao Bio is developing this delivery system with an initial focus on addressing significant unmet needs in oncology and rare monogenic skeletal muscle diseases.
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.
Willow is a female technology company that develops an in-bra wearable breast pump, replacement parts, breastfeeding essentials, pumping bras, cases and bags, breastfeeding essentials, and more.
Janux Therapeutics develops immunotherapies that generate immune responses to prevent tumors and not affecting a patient’s healthy tissue. Janux technology targets all three stages of the anti-tumor immune response.
GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders. GH Research PLC's initial focus is on developing its novel and proprietary 5-MeO-DMT therapies for the treatment of patients with Treatment-Resistant Depression.
Tyra Biosciences, Inc. is a precision oncology company focused on developing purpose-built therapies to overcome tumor resistance and improve outcomes for patients with cancer. Tyra is using its proprietary SNÅP platform, which is optimized to enable rapid and precise refinement of structural design through iterative molecular SNÅPshots, in order to generate next-generation product candidates that are specifically designed to address acquired drug resistance and provide alternative treatment options. Tyra is initially focused on developing a pipeline of selective inhibitors of the Fibroblast Growth Factor Receptor, or FGFR, family, which are altered in approximately 7% of all cancers. Tyra’s lead product candidate, TYRA-300, is designed to selectively inhibit FGFR3, with an initial focus on patients with bladder cancer.
Pyxis Oncology is a developer of antibody therapeutics intended to promote the body's immune response to cancer. The company has engaged in deep analyses of tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and of tumor cell signaling pathways within cold tumors, providing laboratories with development of novel antibody-based immunotherapies. It was founded in 2019 and is based in Boston, Massachusetts.
Omega Therapeutics, Inc. is a development-stage biotechnology company leveraging its proprietary epigenomic programming platform to biologically engineer a new class of programmable epigenetic medicines.
Graphite Bio is a clinical-stage, next-generation gene editing company harnessing high efficiency targeted gene integration to develop a new class of therapies to potentially cure a wide range of serious and life-threatening diseases. Graphite Bio is pioneering a precision gene editing approach that could enable a variety of applications to transform human health through its potential to achieve one of medicine’s most elusive goals: to precisely “find & replace” any gene in the genome. Graphite Bio’s platform allows it to precisely correct mutations, replace entire disease-causing genes with normal genes or insert new genes into predetermined, safe locations.
Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on advancing new therapies for millions of patients living with inflammatory diseases and autoimmune disorders. Ventyx’s clinical stage pipeline includes VTX958, a Phase 1 allosteric TYK2 inhibitor for the treatment of a broad range of autoimmune diseases, VTX002, a Phase 2-ready S1P1 receptor modulator for the treatment of ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is a mediator of multiple inflammatory conditions.
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.
Satsuma Pharmaceuticals is a clinical-stage biopharmaceutical company engaged in developing a novel therapeutic product for the treatment of migraine. Its product candidate, STS101, is a drug-device combination of a dry-powder formulation of dihydroergotamine mesylate, or DHE, which can be self-administered with a pre-filled, single-use, nasal delivery device.
Artiva Biotherapeutics is a private biotech company advancing a pipeline of off-the-shelf, allogeneic NK cell therapies, for the treatment of hematologic cancers or solid tumors. It was founded on the understanding that NK cells hold enormous therapeutic potential as cancer therapies and the dedication and expertise to overcome technological barriers in the scaling and manufacturing of these cells. At Artiva, its mission is to deliver highly effective cell therapies that are also safe and immediately available and accessible to any cancer patient who stands to benefit. Artiva’s platform delivers scalability, quality, and cryopreservation to support a pipeline of safe, effective, versatile, and accessible product candidates.
Centessa Pharmaceuticals is a next-generation biopharmaceutical company that aims to reshape the traditional drug development process. The company applies an asset-centric R&D model at scale to advance a portfolio of highly validated programs led by industry-leading teams. Each program is developed by a Centessa Subsidiary and supported by a centralized infrastructure and the Centessa management team.
Shoreline Biosciences is a biomedical company intended to establish immunotherapies for seriously ill patients. Its proprietary cell therapy technology platform is based on deep expertise in iPSC differentiation methods and genetic programming of the IL-15/CISH pathway for enhanced metabolic fitness and persistence of the engineered NK Cells to improve anti-cancer activity. The company was founded in 2020 and is headquartered in La Jolla, CA, USA.
Design Therapeutics is a biotechnology company developing a new class of therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company’s lead program is focused on the treatment of Friedreich’s ataxia and discovery efforts are ongoing in other degenerative diseases.
Verve Therapeutics is a genetic medicines company pioneering a new approach to the care of cardiovascular disease, transforming treatment from chronic management to single-course gene editing medicines. It has been purpose-built for the task ahead, with a team of experts in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.
Affinivax is a biopharmaceutical company developing vaccines used to enhance the depth of protection against infectious diseases. This novel and highly efficient vaccine conjugation technology – named MAPS for Multiple Antigen Presenting System – has the potential to deliver novel vaccines that offer broader disease coverage than existing vaccines, as well as protect against infectious diseases for which there are no effective immunization strategies today. The company is rapidly advancing a lead program targeting Streptococcus pneumoniae (pneumococcus), a pathogen that is responsible for significant morbidity and mortality in children and adults worldwide. Affinivax was established based on the knowledge and insights of world-leading experts in the fields of infectious diseases and vaccines. The company is backed by an investment from the Bill & Melinda Gates Foundation and has an exclusive license from Boston Children’s Hospital for intellectual property related to the MAPS technology platform.
VISEN is an innovative biopharmaceutical company focused on endocrine diseases. We are dedicated to providing innovative therapies and compassionate, patient-centric care, because we believe that achieving better treatment processes and outcomes results in living better lives. Putting patients’ need first, VISEN is committed to providing first-in-class or best-in-class products and treatments for endocrine diseases. Our therapeutic areas cover endocrine diseases in adults and children, and rare endocrine diseases. VISEN comprises seasoned professionals with multinational pharmaceutical experiences and leverages cutting-edge technologies and leading resources across the world. We are focused on the Chinese market, and have established offices in Shanghai, Beijing, Hong Kong and Taipei. We have also launched our Greater China R&D and manufacturing site in Suzhou to enhance R&D, manufacturing and commercialization efforts. Our goal is to enable Chinese endocrine patients to benefit from the world's most advanced and reliable treatment solutions earlier.
Scorpion Therapeutics is a clinical-stage oncology company specializing in precision medicine for cancer patients. It develops targeted small-molecule drugs to interact with validated cancer targets. It aims to leverage its platform to advance a broad pipeline of wholly-owned, optimized compounds across molecules targeting validated oncogene targets, molecules for previously undruggable targets, and molecules for novel cancer targets.
Biomea Fusion is a privately held precision medicine company with a mission to revolutionize drug development to create more effective therapies for cancer patients. Biomea Fusion has a development portfolio that targets specific gene alterations which occur in the DNA of patients that translate into key drivers of tumor growth. It is Biomea’s goal to move these potentially breakthrough medicines swiftly through the development process and deliver highly impactful treatments for patients in need. The lead program targets the protein-to-protein interaction between menin and the MLL complex for the treatment of various tumors.
Ikena Oncology discovers and develops biomarker-driven therapies for cancer patients. The company focuses on developing novel cancer therapies that drive the formation and spread of cancer. Ikena Oncology provides medical needs by discovering chemistry, translational science, and patient-centric drug development.
IconOVir Bio is a preclinical-stage biotechnology company developing oncolytic virus therapy for the treatment of patients with cancer. Its oncolytic virus platform is designed to address key limitations of first- and second-generation oncolytic viruses to help healthcare professionals provide personalized therapy for cancer patients.
SomaLogic operates as a protein biomarker discovery and clinical diagnostics company. It offers SOMAmers (Slow-Offrate Modified Aptamers), which are modified nucleic acid-based protein-binding reagents that are specific for their cognate protein; and SOMAscan that provides protein detection and equipment. The company’s SOMAmer/SOMAscan technology enables to discover protein biomarker signatures; drug discovery and development; and clinical diagnostics. Its products have applications in the diagnostics of various diseases in oncology, neurology, cardiovascular and metabolic disease, and other diseases and conditions. SomaLogic was founded in 1999 and is headquartered in Boulder, Colorado.
Cullinan Pearl develops an orally available tyrosine kinase inhibitor to target Epidermal Growth Factor Receptor exon 20 mutations. Cullinan Pearl is a subsidiary of Cullinan Oncology.
Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle focused drug discovery platform. Edgewise’s platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases.
Talaris Therapeutics is a operator of a biotechnology company engaged in the development of innovative cellular therapies. The company's proprietary allogeneic cell therapy product has shown to induce tolerance in recipients of solid organ transplants from unmatched and unrelated donors, enabling living donor kidney transplant recipients to wean completely off immunosuppression one year after transplant.
Olema is a preclinical biotechnology company developing new drugs for the treatment and prevention of estrogen receptor (ER) positive breast cancer. The company focuses on the discovery, development, and commercialization of targeted therapies for women's cancers. It leverages the knowledge of the molecular action of the ER to design compounds that are superior to both the current drugs and those in development. Olema leverages their deep understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance with the goal of transforming the standard of care for pre- and post-menopausal women living with cancer by developing more convenient and effective therapies.
Flame Biosciences is a clinical-stage company focused on the discovery, development, and commercialization of transformative therapies for cancer and other inflammatory diseases.
Rain Oncology Inc. is a precision oncology company developing therapies that target oncogenic drivers to genetically select patients it believes will most likely benefit.
Kinnate Biopharma develops precision oncology therapeutics to treat patients with genomically-defined cancers. Its mission is to expand the reach of targeted therapeutics by developing products for underserved populations. The company utilizes its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine. Its programs include candidates in preclinical development for cancers that are driven by specific oncogenic alterations in either the BRAF kinase gene or in the FGFR2 and FGFR3 kinase genes.
Olema is a preclinical biotechnology company developing new drugs for the treatment and prevention of estrogen receptor (ER) positive breast cancer. The company focuses on the discovery, development, and commercialization of targeted therapies for women's cancers. It leverages the knowledge of the molecular action of the ER to design compounds that are superior to both the current drugs and those in development. Olema leverages their deep understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance with the goal of transforming the standard of care for pre- and post-menopausal women living with cancer by developing more convenient and effective therapies.
Cogent Biosciences uses proprietary T-cell engineering technology in combination with tumor-targeting antibodies to activate the body’s own immune system to fight cancer. Unum’s lead program, based on its antibody-coupled T-cell receptor technology, is expected to enter Phase I clinical testing in the coming months to assess safety and efficacy.
Annexon is a clinical-stage biopharmaceutical company developing a pipeline of novel therapies for patients with classical complement-mediated disorders of the body, brain and eye. The company’s pipeline is based on its platform technology addressing well-researched classical complement-mediated autoimmune and neurodegenerative disease processes, both of which are triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. The company’s first product candidate, ANX005, is a full-length monoclonal antibody formulated for intravenous administration in autoimmune and neurodegenerative disorders. The company’s second product candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration for the treatment of neurodegenerative ophthalmic disorders. Annexon is advancing its current programs while evaluating additional orphan and large market indications. Annexon is deploying a disciplined, biomarker-driven development strategy designed to establish that its product candidates are engaging the target at a well-tolerated therapeutic dose in the intended patient tissue.
C4 is building on the knowledge with synthetic chemical compounds which it calls Degronimids. These compounds can be engineered and linked together to bind with many molecular targets that today’s drugs can’t bind with, Cohen said. The key insight, he said, is that the scientific founders have discovered a way to bind with select disease-causing proteins, and flag them as cellular trash, so that the ordinary proteasome garbage disposal system can get rid of them. Selecting the precise disease-related proteins to send to the trash is obviously critical, because any drug like this could cause serious side effects if it sent healthy proteins involved in normal cell processes to the trash. C4 Therapeutics is developing a new class of targeted protein degradation (TPD) therapeutics for the treatment of a broad range of diseases. Our Degronimid™ platform incorporates highly selective small molecule binders to target disease-causing proteins and facilitate their rapid destruction and clearance from the cell through the natural ubiquitin/proteasome system (UPS). Because of this distinctive mechanism, Degronimids are capable of hitting many more targets, including those previously thought to be undruggable, while reducing the potential for drug resistance. The broad applicability of Degronimids, and our chemical biology platform designed for accelerated validation, have the potential to make an unprecedented impact across many diseases through multiple industry collaborations as well as proprietary programs. The Company has received an exclusive worldwide license to the Degronimid platform and related IP from Dana-Farber.
Pliant Therapeutics is a clinical stage biopharmaceutical company focused on discovering and developing novel therapies for the treatment of fibrosis. Pliant's lead product candidate, PLN-74809, is an oral small-molecule dual selective inhibitor of avß6 and avß1 integrins that it is developing for the treatment of idiopathic pulmonary fibrosis, or IPF, and primary sclerosing cholangitis, or PSC. PLN-74809 has received Orphan Drug Designation from the U.S. Food and Drug Administration for both IPF and PSC. Pliant is currently recruiting two Phase 2a trials of PLN-74809 for the treatment of IPF and plans to initiate a Phase 2a trial in PSC in the second half of 2020. Pliant's second product candidate, PLN-1474, is a small-molecule selective inhibitor of avß1 for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis, or NASH, which Pliant has partnered with Novartis. PLN-1474 is currently undergoing a Phase 1 trial. In addition to clinical stage programs, Pliant currently has two preclinical programs targeting oncology and muscular dystrophies.
ALX Oncology is a preclinical stage biotechnology company developing innovative immuno-oncology therapies for cancer. It is an independent, private biotechnology company. The founding of Alexo Therapeutics was based upon technology invented by Aaron Ring, K. Christopher Garcia, Kipp Weiskopf, Aron Levin, and Irving Weissman at Stanford University. This discovery involves soluble versions of signal regulatory protein-α (SIRPα) that have been engineered to bind CD47 with significantly greater affinity than natural SIRPα.1.
Aligos Therapeutics, Inc. is a clinical stage biopharmaceutical company that was founded in 2018 with the mission to become a world leader in the treatment of viral infections and liver diseases. Aligos is focused on the development of targeted antiviral therapies for chronic hepatitis B (CHB) and coronaviruses as well as leveraging its expertise in liver diseases to create targeted therapeutics for nonalcoholic steatohepatitis (NASH). Aligos’ strategy is to harness the deep expertise and decades of drug development experience its workforce has in liver disease, particularly viral hepatitis, to rapidly advance its pipeline of potentially best-in-class molecules.
Avidity Biosciences, Inc. is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has development efforts focused on immune and other cell types.
Nkarta seeks to improve upon the efficacy of cell therapy, making it more potent, better tolerated and more rapidly available to a broad population of patients with a variety of hematologic and solid tumor malignancies. While T-cell therapy, often known as CAR-T, has proven successful in certain cancer indications, its promise has been limited to a few hematologic malignancies and the side effects can be severe. Natural Killer cells are the immune system’s first responders against diseases and pathogens. Unlike T cells, they have an inherent ability to target tumor cells without genetic alteration, and therefore hold significant promise in the development of potent therapies that can be used more readily for diverse tumor types. Nkarta’s differentiated cell therapy approach is designed to boost and enhance the inherent power of Natural Killer cells to target and destroy tumor cells, while also offering a better tolerated safety profile.
Repare Therapeutics is a developer of oncology drugs designed to target specific vulnerabilities of tumor cells. The company's drugs combine a proprietary high throughput gene-editing and target discovery method with high-resolution protein crystallography, computational biology and clinical informatics, enabling patients to improve cancer treatment both with single therapy and in combination with existing drugs and treatments.
Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP) as well as the Orphan Disease Center (ODC). Pursuant to the research collaboration, GTP conducts IND-enabling preclinical work, and Passage Bio is responsible for clinical development, regulatory, manufacturing and commercialization of all product candidates. The ODC is responsible for natural history studies, KOL engagement, and patient advocacy outreach.