RA Capital Management

Imbria Pharmaceuticals, Inc. is a clinical-stage pharmaceuticals company based in Boston, Massachusetts, founded in 2018. The company specializes in developing innovative therapies aimed at addressing cardiometabolic disorders by enhancing or restoring cellular energy production. Imbria's research focuses on the mechanisms underlying these conditions, particularly in areas such as non-obstructive hypertrophic cardiomyopathy, stable angina, and heart failure with preserved ejection fraction. By translating scientific insights into practical treatments, Imbria Pharmaceuticals aims to meet significant unmet medical needs and improve the lives of patients affected by these life-altering disorders. Through its commitment to understanding cellular metabolism and mitochondrial function, the company works to advance healthcare solutions in the cardiometabolic space.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

Bia Energy is focused on providing sustainable and affordable energy solutions to small and medium-sized enterprises (SMEs) and households in Latin America. The company has developed a virtual energy marketing platform that emphasizes efficient energy consumption and optimization. This platform tracks energy usage and offers automatic recommendations for monthly savings, enabling users to reduce costs while adopting sustainable practices. Through its consumer-centric and data-driven approach, Bia Energy aims to empower its customers by enhancing their energy management capabilities and promoting environmentally friendly energy consumption.

ADCendo ApS is a biotechnology company focused on developing innovative anti-cancer drugs through the use of antibody-drug conjugates (ADCs). The company targets novel receptors that are overexpressed in various cancer types, addressing significant unmet medical needs in the field of oncology. Its ADCs aim to provide targeted therapy for patients suffering from aggressive cancers such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, as well as other carcinomas including those of the breast, colon, pancreas, prostate, ovary, and kidney. Through its research and development efforts, ADCendo strives to create effective treatment options for patients facing challenging cancer diagnoses.

Metsera is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for obesity and metabolic conditions. The company focuses on advancing a variety of oral and injectable treatment options, including incretin, non-incretin, and combination therapies. These treatments are designed to address multiple therapeutic targets, positioning Metsera to meet the evolving demands of the weight loss treatment landscape. Additionally, Metsera integrates proprietary health technology tools to provide personalized care, enhancing the effectiveness of its therapeutic offerings.

Innoventric is a medical device company based in Ness-Ziona, Israel, focused on developing innovative percutaneous solutions for Tricuspid Regurgitation and right heart disease. Founded in 2017, the company has made significant advancements with its Trillium™ technology, designed to provide functional tricuspid valve replacement through cross-caval anchoring and a unique multi-valve design. This technology aims to regulate blood flow effectively, thereby enhancing both the lifespan and quality of life for patients. Innoventric has successfully completed over ten implantations of the Trillium™ technology as part of its first clinical trial in Europe and maintains a robust patent portfolio covering its key innovations in tricuspid treatment.

Enara Bio is a biotechnology company based in Oxford, United Kingdom, focused on developing vaccines and immunotherapies for cancer treatment and prevention. Founded in 2016, the company specializes in identifying and utilizing novel antigens derived from endogenous retroviral DNA sequences to create therapeutic cancer vaccines. Enara Bio's technology platform enables the exploration of dark antigens, assessing their tumor specificity and immunogenic potential, which is crucial for effective cancer therapies. The company's innovative approach targets the interaction between T-cells and cancer cells, aiming to create targeted immunotherapies that can benefit a broad patient population.

Aktis Oncology is a biotechnology company focused on developing targeted radiopharmaceuticals for the treatment of various solid tumor cancers. The company has created proprietary platforms that generate tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered to achieve high penetration into tumors and prolonged residence time, allowing for effective tumor elimination while reducing side effects associated with treatment. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing the precision of cancer treatment.

Halda Therapeutics is a research-stage drug discovery company focused on developing next-generation precision medicines. The company utilizes a novel therapeutic modality aimed at creating treatments that can address various disease states. By modifying disease-causing pathways, Halda Therapeutics seeks to enable healthcare providers to offer tailored therapies to patients, enhancing the effectiveness of treatment based on individual patient needs.

IDRx is a clinical-stage biopharmaceutical company focused on developing precision therapies for oncology, specifically targeting gastrointestinal stromal tumors. The company specializes in designing combination therapies that utilize engineered drug combinations to address key tumor escape mechanisms, aiming to enhance treatment efficacy and prolong patient responses. By creating these targeted therapies, IDRx seeks to revolutionize cancer treatment and support healthcare professionals in delivering more effective medicines to patients.

Outpace Bio is a biotechnology company that creates smart cell therapies aimed at improving efficacy and safety. It offers a platform that combines custom-designed biological functions with cellular control modalities that program the right response at the right time and place. It creates a mechanism-driven solution tailor-made to improve efficacy and safety. The company collaborates with Lyell Immunopharma to develop and commercialize a potential immune cell therapy for the treatment of cancer, auto-immune diseases, infectious diseases, degenerative diseases, regenerative medicine, metabolic disorders, and genetic disorders, enabling healthcare providers to cure a wide range of diseases as per patient's need.

Gameto is a biotechnology company focused on improving female reproductive health through innovative therapies. Recognizing that women's health is an underserved area with limited treatment options, Gameto aims to develop advanced solutions that enhance patient outcomes. The company utilizes cellular engineering to create female reproductive cell lines, employing proprietary combinations of transcription factors to reprogram human induced pluripotent stem cells (hiPSCs) into engineered ovarian and endometrial cell lines that function similarly to natural cells. This platform supports a portfolio of cellular therapeutics targeting various female reproductive diseases. Gameto's flagship program, Fertilo, is designed to enhance the processes of in vitro fertilization (IVF) and egg freezing, making them shorter, safer, and more effective. Additional applications of Gameto's technology include Deovo, an organoid of the female reproductive system, and Ameno, a cell-based therapeutic aimed at alleviating the health issues associated with primary ovarian insufficiency and menopause.

AltruBio is a biopharmaceutical company focused on developing targeted antibody therapeutics for cancer and immune-related inflammatory diseases. Established in Delaware with its principal office in the San Francisco Bay Area, AltruBio integrates global resources to advance its research and development initiatives. The company has its roots in Taipei, Taiwan, where it has been working since June 2000 on discovering and developing novel therapeutics to address unmet medical needs. AltruBio employs a sophisticated discovery platform to identify and validate new antibody therapeutics. Currently, the company is developing several potential therapeutic antibodies that target a range of conditions, including autoimmune diseases and various types of cancer, such as pancreatic, stomach, colorectal, ovarian, and lung cancers, at different stages of development.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

ProfoundBio is a clinical-stage biotechnology company dedicated to creating innovative antibody-based therapeutics aimed at treating cancer. Utilizing proprietary technology platforms, the company has developed a diverse pipeline of antibody-drug conjugate (ADC) candidates targeting both solid tumors and hematological malignancies. These drug candidates are currently in various stages of discovery, preclinical, and clinical development. ProfoundBio's focus is on harnessing the immune system to provide patients with more effective treatments that have the potential for curative outcomes.

Nature’s Toolbox is a biotechnology company based in New Mexico that has developed a proprietary host-independent biological manufacturing and development platform. Founded in 2015, the company focuses on identifying, evaluating, and prioritizing naturally occurring drug discovery targets. Its platform provides high-throughput screening capabilities for the rapid prototyping of biological materials, allowing for real-time metabolic profiling of bacteria, fungi, and plants. This technology facilitates the discovery, characterization, and production of bioactive natural products, which can be applied to a range of applications, including protein-based therapies for cancer and autoimmune disorders. By enhancing reproducibility and reducing the resource footprint, Nature’s Toolbox aims to accelerate the drug development process for its clients.

AM Batteries, founded in 2016 and headquartered in Chelmsford, Massachusetts, specializes in the manufacture of lithium-ion batteries utilizing innovative solvent-free electrode manufacturing technology. This approach aims to address the challenges associated with traditional solvent-based processes by eliminating the need for solvent recovery and electrode drying. As a result, AM Batteries' technology not only reduces energy consumption in battery production but also has the potential to enhance battery performance, enabling faster charging and lower production costs. The company's focus on dry electrode manufacturing positions it as a forward-thinking player in the battery industry, committed to improving efficiency and sustainability in battery technology.

Mariana Oncology is a biotechnology company focused on developing radiopharmaceuticals designed to target cancer cells using radioactive drugs. The company leverages expertise in ligand discovery, radiochemistry, and radiobiology, along with oncology translational and clinical research, to create a diverse pipeline aimed at treating various solid tumor types. By advancing the use of radiomedicines, Mariana Oncology seeks to enhance treatment options for medical professionals in their fight against cancer.

Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Based in Newton, Massachusetts, Abcuro creates therapeutic antibodies targeting novel mechanisms related to these diseases. The company utilizes advanced bioinformatics to analyze transcription data from human diseases, which aids in the identification of new therapeutic targets. Through ex-vivo validation in diseased tissues, Abcuro seeks to uncover innovative methods to modulate key components of the immune system. Established in 2015, Abcuro aims to enhance treatment strategies in the healthcare industry by offering effective solutions for complex immunological conditions.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Hemab is a company that creates preventative treatments for bleeding and thrombosis problems. The medicines developed by the business aggregate, modify, balance, and at times recruit endogenous clotting components already present in the blood to the site of injury, allowing patients to produce hemostatic plugs or clots to avert life-threatening complications. Hemab Therapeutics was established in 2017 in Copenhagen, Hovedstaden by Johan H. Faber and Mads Behrndt.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company focuses on developing and commercializing innovative silk biomaterial technologies. Its key product, the MIMIX microneedle platform, facilitates the transdermal delivery of vaccines and therapeutics. This sustained release patch technology allows for a controlled release of vaccines, mimicking the natural infection process to enhance immune response and effectiveness. Vaxess's patch is engineered for stability, eliminating the need for refrigeration, which enables shipping and application in low-resource settings. In addition to its vaccine delivery systems, Vaxess is also engaged in creating novel therapies aimed at oncology care and infectious diseases, ultimately striving to improve patient outcomes with reduced treatment burdens.

Apogee Therapeutics is a biotechnology company based in San Francisco, California, founded in 2022. The company focuses on developing differentiated biologics for the treatment of immunological and inflammatory disorders, specifically targeting atopic dermatitis and chronic obstructive pulmonary disease. Apogee Therapeutics aims to address high unmet medical needs by advancing novel therapies that utilize advanced antibody engineering to enhance therapeutic properties such as half-life. The company's key programs, APG777 and APG808, are designed to leverage established mechanisms of action to improve treatment outcomes for patients suffering from these conditions.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company focuses on developing and commercializing innovative silk biomaterial technologies. Its key product, the MIMIX microneedle platform, facilitates the transdermal delivery of vaccines and therapeutics. This sustained release patch technology allows for a controlled release of vaccines, mimicking the natural infection process to enhance immune response and effectiveness. Vaxess's patch is engineered for stability, eliminating the need for refrigeration, which enables shipping and application in low-resource settings. In addition to its vaccine delivery systems, Vaxess is also engaged in creating novel therapies aimed at oncology care and infectious diseases, ultimately striving to improve patient outcomes with reduced treatment burdens.

Rivus Pharmaceuticals is a biopharmaceutical company focused on enhancing cardio-metabolic health through the development of controlled metabolic accelerators (CMAs). These innovative therapeutics aim to improve cellular metabolism and target the root causes of prevalent metabolic and cardiovascular diseases. By providing oral small-molecule treatments, Rivus addresses critical metabolic risk factors associated with cardiovascular mortality and morbidity, thereby contributing to better health outcomes for patients suffering from these conditions.

Empatica Inc. is a company that specializes in developing wearable medical sensor devices aimed at health monitoring, treatment, and research. Founded in 2013 and based in Cambridge, Massachusetts, Empatica's offerings include the E3 wristband, which monitors physiological signals in real-time, along with a mobile application and a web dashboard designed for stress and sleep monitoring. The company employs a combination of biosensors and artificial intelligence to analyze human physiology, providing valuable insights for patients, clinicians, and researchers. Its client base includes notable organizations such as Microsoft Research, Sony, University of Trento, and Intel.

MMI S.r.l. is an Italian company that specializes in the manufacture and development of advanced micro instruments designed for surgical interventions. Founded in 2015 and based in Calci, Italy, MMI focuses on creating wristed micro instruments and their associated fabrication processes. The company also offers a proprietary robotic platform that enhances the capabilities of these instruments, facilitating complex reconstruction procedures following traumatic injuries and tumor removals in areas such as the breast, head, and bones. MMI aims to support surgeons globally by providing innovative tools that improve surgical outcomes while also reducing costs for patients and healthcare systems.

Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.

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, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Incyclix Bio is a biotechnology company based in Durham, North Carolina, focused on the development of small molecule inhibitors targeting cyclin-dependent kinases (CDKs) for the treatment of advanced and resistant cancers. Founded in 2020 by a team including Patrick Roberts, Jay Strum, John Bisi, and Fred Eshelman, the company aims to provide potent and selective inhibition of challenging CDK targets. This approach enables pharmaceutical companies and medical practitioners to access innovative therapeutic options, thereby enhancing treatment opportunities for cancer patients facing difficult cases. Through its research and development efforts, Incyclix Bio seeks to contribute to the advancement of cancer treatment solutions.

Plexium, Inc. is a biotechnology company based in San Diego, California, that specializes in developing targeted protein degradation therapies aimed at treating cancer and neurodegenerative diseases. Founded in 2017, Plexium utilizes a platform that identifies small molecules capable of modulating E3 ligases, which play a crucial role in protein degradation. This platform includes the DELPhe screening technology, designed for high-content readouts from cell-based assays. By harnessing E3 ligases, Plexium's approach enables selective targeting of various cancers, including small cell lung and breast tumors. The company's innovative research extends beyond existing therapies, exploring new drug-like protein degraders and molecular glues to enhance treatment options for cancer and inflammatory diseases.

Electra Therapeutics is a clinical-stage biotechnology company focused on developing therapies that target signal regulatory proteins (SIRP) for treating immunological diseases and cancer. The company has one program in clinical development and two additional preclinical programs. Its lead product candidate, ELA026, is a monoclonal antibody designed to target SIRP on the surface of myeloid and T cells, effectively depleting pathological immune cells. ELA026 is currently being investigated for secondary hemophagocytic lymphohistiocytosis (sHLH), a severe hyperinflammatory condition for which no approved treatment exists.

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

Third Harmonic Bio is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for severe allergies and inflammation. The company is focused on creating first-in-class oral small-molecule inhibitors targeting the KIT receptor, which plays a crucial role in the survival and function of mast cells—key immune cells involved in allergic responses. Its lead product candidate, THB001, is designed to provide selective inhibition of KIT, while another candidate, THB335, aims to address multiple mast cell-driven inflammatory diseases affecting the skin, respiratory system, and gastrointestinal tract. Through its research and development efforts, Third Harmonic Bio seeks to advance the management of inflammatory diseases, enhancing patient outcomes and quality of life.

AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.

Tasso, Inc., established in 2012 and based in Seattle, Washington, specializes in manufacturing clinical-grade, at-home blood sample collection devices. Their innovative product allows patients to conveniently and painlessly collect their own blood at home using a simple, push-button mechanism, and then mail the samples to a qualified lab for analysis. This approach aims to enhance accessibility and comfort in blood-based diagnostics, reducing the need for clinic visits.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Antios Therapeutics, Inc. is a biopharmaceutical company based in Atlanta, Georgia, founded in 2018. The company focuses on developing innovative therapies for viral diseases, particularly through its oral drug candidate, ATI-2173, aimed at treating patients infected with Hepatitis B and Hepatitis D viruses. With a leadership team that possesses extensive experience in the field, Antios Therapeutics is dedicated to advancing its research and development efforts toward potentially curative treatments for Hepatitis B virus infections.

Cedilla Therapeutics, Inc. is focused on developing targeted small-molecule medicines aimed at treating cancer and other diseases resulting from protein dysregulation. The company leverages unique insights into protein stability to create innovative therapeutic approaches, including direct ligand-induced degradation, disruption of protein-protein interactions, and the identification of upstream regulators that influence protein stability. Cedilla’s technology allows for the mapping of protein susceptibility to enhance endogenous degradation pathways, facilitating the transition from stable to susceptible protein states. Founded in 2018 and headquartered in Cambridge, Massachusetts, Cedilla Therapeutics aims to broaden the applicability of its therapies beyond oncology, addressing a range of conditions associated with protein dysregulation.

Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that have the potential to treat a wide range of severe health conditions. By leveraging its innovative technology, Expansion Therapeutics aims to provide effective treatment options for patients suffering from these challenging diseases.

Obsidian Therapeutics develops innovative cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Founded in 2015 and based in Cambridge, Massachusetts, the company focuses on creating next-generation therapies that utilize pharmacologic operating systems to provide precise control over protein activity within cells. This technology allows for the development of adoptive cell therapies with advanced functionalities that can be managed by physicians using simple, safe, and orally active medications. By offering improved control over treatment, Obsidian Therapeutics aims to provide better outcomes for patients compared to existing cell therapy options.

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.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that focuses on drug discovery through its innovative platform targeting solute carrier (SLC) transporters. Established in 2016, the company aims to improve patient outcomes by developing small molecule therapies for various diseases, including those related to immunometabolism, lysosomal function, and mucosal defense. By utilizing advanced chemoproteomics techniques, Jnana accelerates the identification of therapeutic targets, addressing significant unmet medical needs in areas such as immuno-oncology, inflammatory disorders, and neurological diseases. The company's approach seeks to enhance drug discovery efficiency by comprehensively targeting the vast family of membrane proteins associated with SLC transporters.

Sydnexis is focused on developing a proprietary treatment for pediatric progressive myopia, addressing a significant global medical need. The company manufactures a topical eyedrop formulation specifically designed to treat the progression of myopia in children. This innovative approach enables healthcare professionals to effectively manage and reduce the incidence of eye disorders in pediatric patients.

T-knife is a biotechnology company focused on developing T cell receptor therapies aimed at treating cancer. Utilizing a humanized T cell receptor (TCR) mice platform, the company is capable of carrying human TCRαβ gene loci and recombining a diverse array of human TCRs. This innovative approach facilitates the efficient generation and virtual selection of T cell receptors tailored for specific human tumor antigens, enhancing the potential for effective cancer therapies. Through its advanced technology, T-knife aims to improve treatment options for patients battling cancer.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.

Synthekine Inc. is a biotechnology company focused on developing innovative cytokine therapies and immunotherapies for cancer and autoimmune disorders. Established in 2018 and located in Menlo Park, California, the company employs advanced platforms such as engineered partial agonists and orthogonal cell therapies. Its Synthekine Platform utilizes surrogate agonists instead of traditional mutant cytokines, allowing for the creation of therapeutics that enhance efficacy while minimizing side effects. The product pipeline includes STK-009, an orthogonal ligand, and SYNCAR-001, a CAR-T therapy targeting CD-19, along with STK-012, which consists of partial agonists of IL-2. Synthekine aims to revolutionize treatment paradigms by harnessing the principles of cytokine partial agonism and immunological specificity, leading to novel immunotherapies that improve patient outcomes.

Bright Peak Therapeutics is a biotechnology company focused on developing immunotherapies for cancer and autoimmune diseases. The company specializes in cytokine therapeutics that enable tissue and cell-specific targeting of cytokine payloads. This approach aims to enhance the efficacy of treatments, ultimately improving patient outcomes and quality of life. By leveraging innovative immuno-cytokines, Bright Peak seeks to provide more effective therapeutic options for complex medical conditions.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, that specializes in developing therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating innovative treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact millions of individuals. Eliem utilizes its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, including its lead candidate, ETX-155, aimed at addressing major depressive disorder and focal onset seizures. The company's mission centers on meeting unmet medical needs and improving patients' quality of life through its novel therapies.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Forge Biologics, Inc. is a contract development and manufacturing organization based in Columbus, Ohio, focused on developing and manufacturing gene therapies and genetic medicines. The company specializes in viral vector gene therapies aimed at treating genetic diseases, including a novel approach combining adeno-associated virus (AAV) with umbilical cord transplants for infantile Krabbe disease, a severe neurodegenerative condition. Founded in 2019, Forge Biologics partners with scientists, healthcare professionals, and biotech and pharmaceutical companies to facilitate the transition of gene therapy programs from preclinical development to clinical and commercial-scale production. By providing end-to-end capabilities, the company aims to accelerate the availability of transformative medicines for patients suffering from rare genetic disorders.

Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at generating immune responses to combat tumors while preserving healthy tissue. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux targets all three stages of the anti-tumor immune response. The company's approach is designed to enhance the efficacy of cancer treatments by specifically engaging the immune system to identify and eliminate cancer cells.

Antios Therapeutics, Inc. is a biopharmaceutical company based in Atlanta, Georgia, founded in 2018. The company focuses on developing innovative therapies for viral diseases, particularly through its oral drug candidate, ATI-2173, aimed at treating patients infected with Hepatitis B and Hepatitis D viruses. With a leadership team that possesses extensive experience in the field, Antios Therapeutics is dedicated to advancing its research and development efforts toward potentially curative treatments for Hepatitis B virus infections.

GH Research PLC is a clinical-stage biopharmaceutical company that aims to transform the treatment of psychiatric and neurological disorders. The company is primarily focused on developing novel and proprietary therapies based on 5-MeO-DMT, a compound known as Mebufotenin, to address the needs of patients suffering from Treatment-Resistant Depression (TRD). Through its innovative approach, GH Research seeks to provide new therapeutic options for individuals who have not responded to conventional treatments.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Tyra Biosciences is a clinical-stage biotechnology company based in Carlsbad, California, that specializes in developing small molecule therapies for cancer treatment. Founded in 2018, the company aims to address drug resistance in cancer therapies through its proprietary SNÅP platform, which facilitates rapid and precise drug design by generating iterative molecular snapshots. Tyra is particularly focused on creating selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. The company's lead product candidate, TYRA-300, specifically targets FGFR3 and is initially aimed at patients with metastatic urothelial carcinoma of the bladder and urinary tract. Through its innovative approach, Tyra Biosciences seeks to improve treatment outcomes for cancer patients by providing alternatives to existing therapies.

Pyxis Oncology, established in 2019 in Boston, Massachusetts, is a biotechnology company specializing in the development of antibody therapeutics for cancer treatment. The company focuses on promoting the body's immune response to cancer by analyzing tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and tumor cell signaling pathways within cold tumors. Pyxis aims to create novel antibody-based immunotherapies to directly kill tumor cells and address underlying pathologies that enable cancer proliferation and immune evasion, with the goal of improving patient outcomes for difficult-to-treat cancers.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.

Day One Biopharmaceuticals is a clinical-stage biotechnology company that specializes in developing targeted cancer therapies for patients of all ages, with a particular focus on pediatric patients. Recognizing the advancements in cancer research, the company aims to create treatments that can benefit both children and adults diagnosed with genetically defined cancers. Day One Biopharmaceuticals prioritizes the rapid development of therapies to meet the urgent needs of families facing cancer diagnoses. Their lead product candidate, DAY101, is an oral, highly-selective pan-RAF kinase inhibitor designed to penetrate the brain, addressing a significant treatment gap in pediatric oncology. By licensing and acquiring promising products from research institutions and other companies, Day One Biopharmaceuticals strives to bring effective medicines to market for those impacted by cancer.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

IO Biotech is a clinical-stage biopharmaceutical company focused on developing innovative immune therapies for cancer treatment. Utilizing its proprietary T-win technology platform, the company creates immune-modulating cancer vaccines that activate T cells specifically targeting immune-suppressive molecules. This approach aims to induce the immune system to disrupt multiple pathways responsible for tumor-induced immunosuppression. IO Biotech has a solid track record in advancing both preclinical and clinical compounds, with its two lead candidates targeting IDO and PD-L1 currently in clinical development, while several additional therapies remain in the preclinical stage.

Werewolf Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapeutics that enhance the body’s immune response to cancer. Founded in 2017, the company utilizes its proprietary PREDATOR platform to engineer conditionally activated molecules, known as INDUKINE molecules. These therapeutics are designed to remain inactive in peripheral tissues but activate selectively within the tumor microenvironment, addressing the limitations of traditional proinflammatory immune therapies. By stimulating both adaptive and innate immunity, Werewolf Therapeutics aims to improve cancer treatment outcomes.

Kira Pharmaceuticals is a biotechnology company founded in 2017 and based in Suzhou, China. It specializes in developing complement-targeted therapies aimed at treating immune-mediated diseases. The company focuses on pioneering drug discovery related to the complement system, with the goal of providing transformative therapies for individuals suffering from complement-mediated conditions.

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.

Acumen Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing therapies for Alzheimer's disease and related neurodegenerative conditions. The company is advancing a targeted immunotherapy drug candidate, ACU193, which is designed to selectively target amyloid-beta oligomers, believed to be a key factor in the progression of Alzheimer's disease. Acumen employs a sensitivity assay to measure soluble amyloid-beta oligomer levels in cerebrospinal fluid, which aids in the enrollment of patients for clinical trials. The Phase 1 clinical trial for ACU193 began in 2021, aiming to assess its safety, tolerability, and pharmacokinetics in patients with mild cognitive impairment and mild dementia resulting from Alzheimer's disease. Founded in 1996 and based in Livermore, California, Acumen Pharmaceuticals is committed to addressing the challenges posed by Alzheimer’s disease through innovative therapeutic approaches.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

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 biopharmaceutical company dedicated to advancing research and development in psychedelic and medicinal mushrooms. It focuses on creating safe and effective therapeutics for mental health issues, with an emphasis on psilocybin-based products. The company is actively launching these products in jurisdictions where they are permitted and is engaged in clinical studies across North America and other regions. Through strategic partnerships with academic and institutional entities, Cybin seeks to develop novel compounds and delivery mechanisms, aiming to provide innovative treatments for various psychiatric and neurological conditions.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

Pandion Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for patients with autoimmune diseases and inflammatory conditions. The company's lead product candidate, PT101, is an engineered variant of interleukin-2 currently undergoing Phase 1a clinical trials for moderate-to-severe ulcerative colitis and systemic lupus erythematosus. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and PT001 and PT002, which are bifunctional molecules designed for tissue-selective immunomodulation in the gastrointestinal tract. These molecules utilize a unique approach to achieve localized treatment, potentially enhancing efficacy and safety compared to traditional therapies. Pandion also collaborates with Astellas Pharma to develop locally acting immunomodulators targeting autoimmune diseases of the pancreas. The company is headquartered in Watertown, Massachusetts.

iTeos Therapeutics is a biopharmaceutical company dedicated to developing immuno-oncology therapies for cancer patients. Founded in 2011, the company operates from headquarters in Cambridge, Massachusetts, and Gosselies, Belgium. iTeos focuses on discovering and advancing small molecule immunomodulators that target the tumor microenvironment, aiming to enhance the body's immune response against cancer. Its pipeline includes EOS-850, a small molecule antagonist of the adenosine A2a receptor, and EOS-448, an antagonist of TIGIT, both currently in Phase 1/2a clinical trials. iTeos' approach is built on the expertise of Ludwig Cancer Research, aiming to improve clinical outcomes by restoring the immune system's ability to fight cancer.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing implanted drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company utilizes a refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of its formulation of the anti-epileptic drug valproic acid, known as CT-010. This innovative approach aims to bypass the blood-brain barrier, enhancing treatment outcomes for patients with chronic neurological conditions. Founded in 2010, Cerebral Therapeutics is dedicated to improving the lives of individuals affected by uncontrolled neurological diseases.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cells for the treatment of cancer and autoimmune diseases. The company aims to enhance the efficacy of cell therapy by leveraging the inherent ability of NK cells to identify and eliminate abnormal cells without the need for genetic alteration. This approach allows for a broader application across various hematologic and solid tumor malignancies while potentially minimizing side effects associated with traditional T-cell therapies. Nkarta combines its NK expansion platform technology with proprietary cell engineering methods to produce a substantial supply of NK cells, enhancing their ability to recognize therapeutic targets and improving their persistence in the body for sustained activity. Through this innovative strategy, Nkarta seeks to provide more potent, well-tolerated, and rapidly available therapies for patients facing diverse cancer types.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, focused on developing patient-specific immunotherapies to treat cancer. Founded in 2016, the company creates personalized vaccines and T cell therapies that harness the immune system to target and eliminate cancer cells. By identifying truncal tumor neo-antigens and other cancer-specific markers, Achilles Therapeutics aims to develop next-generation therapies that selectively attack tumors while sparing healthy tissues. Currently, the company is conducting two clinical trials: the CHIRON trial for patients with non-small-cell lung cancer and the THETIS trial for those with recurrent or metastatic melanoma.

Freenome, Inc. is an artificial intelligence genomics biotech company based in South San Francisco, California, founded in 2014. The company focuses on developing accurate, accessible, and non-invasive blood tests for the early detection of cancer and treatment selection. Utilizing a proprietary multi-omics platform, Freenome employs advanced algorithms to analyze cell-free genomic data, which aids in identifying early-stage cancers and other diseases. This innovative approach aims to transform disease management by enabling timely interventions and proactive treatment strategies. In addition to its diagnostic solutions, Freenome also offers clinical research services to further advance cancer detection and management.

Satsuma Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing and commercializing therapeutics for rare genetic disorders of hemoglobin, including sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing its lead product candidate, IMR-687, which is a highly selective small molecule inhibitor of PDE9. IMR-687 is designed as an oral, once-a-day treatment that aims to modify disease through a multimodal mechanism of action affecting various cell types, including red and white blood cells. Imara is dedicated to addressing the unmet medical needs of patients suffering from these serious hemoglobinopathies.

Attune Pharmaceuticals, Inc. is a biotechnology research and development company based in New York, established in 2015. The company specializes in discovering and developing novel orally administered small molecule therapeutics aimed at treating rare diseases. Its focus includes the creation of heterocyclic derivative compounds and pharmaceutical compositions that address conditions such as hereditary angioedema, thereby providing clinicians with effective treatment options for rare medical conditions.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

SeLux Diagnostics, Inc. is a biotechnology company based in Charlestown, Massachusetts, founded in 2014. It specializes in developing a next-generation phenotyping platform for antibiotic susceptibility testing and diagnostics of infectious diseases. The company utilizes a synthetic amplifier technology that enhances the speed and complexity of antibiotic susceptibility testing, facilitating quicker and more accurate selection of personalized antimicrobial therapies for patients. By streamlining the identification of effective treatments, SeLux Diagnostics aims to improve patient care, reduce hospital stays, and tackle the growing issue of antibiotic resistance by minimizing the reliance on broad-spectrum antibiotics.

WhiteSwell Limited is a Galway-based company focused on advancing the treatment of acute decompensated heart failure (ADHF), a condition that leads to significant hospitalizations and emergency room visits. Founded in 2014, WhiteSwell has developed a unique catheter-based medical device that targets the lymphatic system, enabling the removal of excess fluid from both interstitial and intravascular spaces without compromising renal function. By creating a low-pressure zone at the thoracic duct outlet, the device reduces resistance to lymphatic drainage, thereby improving clinical outcomes for patients suffering from this serious condition. The company also has offices in Shefayim, Israel, and Palo Alto, California, emphasizing its commitment to addressing the critical need for effective and safe treatment options for ADHF, which is associated with high mortality rates.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Xenikos is focused on developing innovative immunotherapy treatments aimed at addressing serious immune diseases and complications arising from transplants. The company specializes in anti-T-cell antibodies that can effectively reset the immune system. This therapeutic approach involves injecting a specific agent that targets and eliminates adult T cells, particularly those that are activated, thereby enhancing treatment outcomes for patients. By leveraging this unique mechanism, Xenikos seeks to restore health and improve the quality of life for individuals facing significant immune-related challenges.

Eidos Therapeutics, Inc. is a clinical stage biopharmaceutical company based in San Francisco, California, that specializes in developing treatments for diseases related to transthyretin (TTR) amyloidosis. The company is advancing AG10, an orally administered small molecule currently in phase 3 clinical trials, which aims to stabilize tetrameric TTR and interrupt the molecular processes leading to amyloidosis. Founded in 2013, Eidos Therapeutics operates as a subsidiary of BridgeBio Pharma, which focuses on creating genetically targeted therapies designed to address the underlying causes of various diseases. Through its innovative approach, Eidos Therapeutics seeks to provide potentially disease-modifying therapies to meet the significant medical needs associated with TTR-related conditions.

Crinetics Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company headquartered in San Diego, California, dedicated to the discovery, development, and commercialization of innovative therapeutics for rare endocrine diseases and endocrine-related tumors. The company’s lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist currently undergoing Phase I clinical trials aimed at treating acromegaly and neuroendocrine tumors. In addition to paltusotine, Crinetics is developing several other therapeutics, including CRN01941, an oral selective nonpeptide sst2 biased agonist for neuroendocrine tumors, and an oral somatostatin type 5 receptor agonist for congenital hyperinsulinism. The company is also working on an oral adrenocorticotrophic hormone antagonist for conditions such as Cushing’s syndrome and congenital adrenal hyperplasia. Founded in 2008, Crinetics Pharmaceuticals is focused on leveraging its internal discovery efforts to create effective treatments while utilizing hormonal biomarker endpoints to enhance the efficiency and cost-effectiveness of its clinical studies.