Novartis

Granite Bio is a biotechnology company focused on developing novel monoclonal antibodies aimed at treating inflammatory, autoimmune, and fibrotic disorders. The company specializes in creating therapeutic antibodies that deplete pathogenic cells and selectively target key biological processes associated with these diseases. By concentrating on these advanced treatments, Granite Bio contributes to the broader fields of biotechnology, pharmaceuticals, and diagnostics, ultimately seeking to improve patient outcomes in complex medical conditions.

Anthos Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapies for cardiovascular and metabolic diseases. Founded in 2018, the company specializes in treatments targeting Factor XI and XIa, essential components of the intrinsic coagulation pathway. Its lead product, MAA868, is an antibody designed to provide an anti-thrombotic effect, allowing healthcare professionals to prevent various thrombotic diseases while minimizing the risk of bleeding. By advancing long-acting treatment options, Anthos Therapeutics aims to improve care for patients suffering from cardiovascular conditions, representing a significant advancement over conventional therapies.

Borealis Biosciences is a discovery-stage biotechnology company focused on developing next-generation RNA-based medicines specifically for kidney diseases. The company utilizes its expertise in molecular and cellular biology, chemistry, and analytical capabilities to identify target opportunities within different patient subsets affected by kidney conditions. By refining methods for delivering RNA medicines to specific cell types, Borealis Biosciences aims to tackle significant unmet medical needs in the treatment of kidney diseases.

Dren Bio is a biotechnology company that specializes in protein engineering technologies designed to target and deplete pathogenic cells, protein aggregates, and other disease-causing agents. With a focus on hematologic neoplasms, solid cancers, autoimmune disorders, and diseases related to protein aggregation, the company aims to provide innovative solutions for healthcare professionals in their efforts to combat various serious diseases. Operating under a holding company model, Dren Bio develops multiple projects as standalone assets, seeking to create value through its diverse portfolio of biotechnology initiatives.

Enterprise Therapeutics, established in 2014 and headquartered in Brighton, UK, is a drug discovery company focused on developing therapies for respiratory diseases. Its primary focus is on chronic obstructive pulmonary diseases, cystic fibrosis, and asthma. The company aims to create novel treatments that target the underlying mechanisms of mucus congestion, reducing lung infections and enhancing patients' quality of life.

Calypso Biotech SA is an immunotherapy biotechnology company based in Plan-les-Ouates, Switzerland, founded in 2013 as a spin-off from Merck Serono. The company specializes in discovering and developing monoclonal antibodies aimed at treating immune pathologies, particularly for conditions with significant unmet medical needs, including severe gastrointestinal diseases. Calypso Biotech operates as a semi-virtual organization, leveraging a highly experienced management team with expertise in drug development. The company focuses on creating substantial value for investors through a portfolio of unique proprietary antibody candidates designed to control homeostasis and improve treatment outcomes for patients.

SanReno Therapeutics specializes in the development, manufacturing, and commercialization of therapies for kidney diseases in several regions including the People's Republic of China, Hong Kong, Macau, Taiwan, and Singapore. Established through a joint venture involving Chinook Therapeutics, Frazier Healthcare Partners, and Pivotal bioVenture Partners China, the company is dedicated to researching and producing innovative biotech solutions aimed at improving the quality of life for patients suffering from kidney-related conditions.

AstronauTx is a biotechnology company focused on developing treatments for Alzheimer's disease and other neurodegenerative conditions. The company aims to restore the normal functioning of astrocytes, which are crucial cells in the brain that support neuronal activity, metabolism, and the integrity of the blood-brain barrier. In conditions such as dementia, astrocytes can become dysfunctional, leading to neurotoxicity and exacerbating disease progression. By targeting astrocyte homeostasis, AstronauTx seeks to enable healthcare professionals to effectively address the underlying mechanisms of neurotoxicity associated with these diseases, ultimately improving treatment options for patients suffering from dementia and related disorders.

Hyku is a biotechnology company focused on developing small molecule therapeutics that target disease-causing proteins through precise covalent binding. Utilizing its innovative platform, Hyku identifies molecules that covalently bind to non-cysteine amino acids, facilitating the creation of novel therapies. These therapies aim to address significant challenges in drug development, including issues related to selectivity, druggability, and resistance. By pushing the boundaries of covalent medicine, Hyku seeks to create effective treatments that can improve patient outcomes and advance the field of therapeutic development.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

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.

Flare Therapeutics is a biotechnology company focused on developing small molecule medicines by exploring the biology of transcription factors. The company aims to identify 'switch sites,' which are druggable regions crucial for regulating transcription factors, to target mutations associated with various diseases. Flare's innovative approach has advanced its drug discovery efforts, resulting in a promising pipeline of programs that primarily address well-established transcription factors. Initially concentrating on precision oncology, the company's research also holds potential applications in neurology, rare genetic disorders, immunology, and inflammation. By leveraging insights from its scientific founders, Flare Therapeutics seeks to transform the treatment landscape for cancer patients and other disease areas.

Orionis Biosciences is a biotechnology company focused on early-stage drug discovery and development, particularly targeting oncology and immunotherapies. Headquartered in Waltham, Massachusetts, with additional research facilities in Ghent, Belgium, the company utilizes a range of innovative technologies to create conditionally active drug modalities aimed at treating diseases with significant unmet medical needs. By inducing molecular proximity and cooperativity, Orionis enhances drug potency and precision, allowing for novel target access. Their approach facilitates the development of a diverse pipeline of drug candidates, including therapies that engage both adaptive and innate immune systems. The company collaborates with strategic partners, including VIB in Belgium, and is supported by a team of seasoned entrepreneurs and scientists, as well as a transatlantic network of investors.

Splice Bio is a biotechnology company based in Barcelona, Spain, focused on developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012 and originally named Proteodesign, S.L., the company aims to address the needs of patients suffering from incurable genetic diseases. The intein platform, developed from research in the Muir Lab at Princeton University, seeks to overcome two significant limitations of traditional adeno-associated virus (AAV) therapies: it enhances the capacity for larger gene delivery and broadens the range of tissues that can be targeted for treatment.

ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells specifically designed to target and treat solid tumors. By utilizing advanced technology, the company aims to create chimeric antigen receptor (CAR) T-cell therapies that exhibit tumor specificity, allowing them to induce cytotoxicity only when interacting with tumor cells while sparing healthy tissues. This selective targeting mitigates the safety risks commonly associated with existing CAR-T therapies, providing a promising approach to cancer treatment that enhances efficacy and reduces potential harm to noncancerous cells.

XOMA Corporation is a biotechnology company focused on the discovery and development of therapeutic candidates, primarily in the fields of monoclonal antibodies and innovative drug technologies. With a robust pipeline, the company is developing proprietary product candidates such as X213, an allosteric inhibitor of prolactin action; XMetA, an insulin receptor-activating antibody for Type 2 diabetes; and therapies targeting metastatic melanoma and renal cell carcinoma. XOMA also has initiatives aimed at addressing unmet medical needs related to hyperparathyroidism. In addition to its own product development, the company licenses antibody discovery and optimization technologies and collaborates with major pharmaceutical firms, including Novartis and Takeda, to enhance clinical development efforts. Founded in 1981 and headquartered in Emeryville, California, XOMA is positioned at the forefront of biopharmaceutical innovation, contributing to advancements in healthcare.

Lightship is a company founded in 2018 that focuses on enhancing access, choice, and equity in clinical trials through decentralized and hybrid clinical services. By leveraging innovations in digital health and technology, Lightship aims to transform the clinical research landscape by improving patient experiences, retention, and trial diversity. The company moves beyond traditional research centers to make clinical studies more accessible, allowing medical research organizations to overcome the barriers and inefficiencies associated with conventional clinical trial models. Its mission centers on placing patients at the forefront, ensuring that clinical research is designed to meet the needs of diverse populations globally.

Exo Therapeutics, Inc. is a drug discovery and development company focused on addressing intractable pharmaceutical targets. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes its proprietary ExoSight platform to develop a pipeline of drug candidates. These candidates are designed to bind to exosites and unique binding pockets, allowing for the reprogramming of enzyme activity to achieve targeted therapeutic effects. This innovative approach aims to provide robust treatments in areas such as oncology and inflammation, while also minimizing potential side effects for patients. By employing a precise and selective methodology, Exo Therapeutics is positioned to unlock new therapeutic possibilities for a variety of diseases.

Immunitas Therapeutics, Inc. is focused on developing targeted therapies for cancer patients through innovative single-cell analyses. Founded in 2019 and based in Cambridge, Massachusetts, the company utilizes a single-cell sequencing platform to explore the biology of immune cells within human tumors. This approach allows Immunitas to identify novel drug targets rooted in human biology and create key biomarkers that assist in selecting patients who may benefit from their therapies. By translating laboratory findings into clinical applications, Immunitas aims to address longstanding challenges in oncology and improve treatment options for complex cancers. The company is advancing multiple programs toward early human studies, leveraging expertise in antibody discovery and engineering to develop effective treatments.

Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2015, the company is dedicated to creating first-in-class therapies aimed at selectively killing cancer cells. Artios's product pipeline features several promising programs, including a focus on DNA polymerase theta (Polθ), which plays a crucial role in DNA repair processes. Additionally, the company is advancing an in-licensed program that targets a newly identified protein linked to DDR and is developing a novel treatment aimed at DNA nucleases involved in these pathways. Artios collaborates with Cancer Research Technology and prominent DNA repair researchers globally to enhance its research and development efforts.

Cellerys, headquartered in Zurich, Switzerland, is a biotechnology company focused on developing a novel therapy for multiple sclerosis (MS). Their proprietary platform aims to induce immune tolerance in MS patients by coupling autologous red blood cells with specific peptides, a process designed to potentially treat MS through antigen-specific tolerance induction. The company's semi-automatic good manufacturing practice process streamlines this procedure, facilitating its application in clinical settings and advancing the care of autoimmune disease patients.

Soteria Biotherapeutics, Inc., founded in 2018 and based in San Francisco, California, focuses on developing innovative immunotherapies for cancer treatment. The company is creating a new generation of conditionally active bispecific T-cell engaging antibodies specifically aimed at treating solid tumors. Utilizing its advanced T-LITE™ platform, Soteria enables small molecule-dependent activation of these bispecific antibody therapies, which facilitates safer and more effective treatment options. This approach allows for pulsatile activity, resulting in reduced side effects and the potential for higher dosing, thereby improving the overall therapeutic experience for patients.

SomaLogic, Inc. is a commercial-stage company based in Boulder, Colorado, specializing in protein biomarker discovery and clinical diagnostics. Founded in 1999, the company has developed advanced technologies such as SOMAmers, slow off-rate modified aptamers that specifically bind to target proteins, and the SOMAscan platform, which facilitates comprehensive protein measurement. This innovative approach allows for the analysis of protein biomarker signatures, significantly enhancing the understanding of health and disease beyond traditional genomic methods. SomaLogic's products are utilized in a variety of applications, including diagnostics for conditions in oncology, neurology, and cardiovascular and metabolic diseases, as well as in drug discovery and development. The company also offers diagnostic tests and protein measurement solutions for life science and biopharma researchers, contributing to advancements in medical science.

Exo Therapeutics, Inc. is a drug discovery and development company focused on addressing intractable pharmaceutical targets. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes its proprietary ExoSight platform to develop a pipeline of drug candidates. These candidates are designed to bind to exosites and unique binding pockets, allowing for the reprogramming of enzyme activity to achieve targeted therapeutic effects. This innovative approach aims to provide robust treatments in areas such as oncology and inflammation, while also minimizing potential side effects for patients. By employing a precise and selective methodology, Exo Therapeutics is positioned to unlock new therapeutic possibilities for a variety of diseases.

Faze Medicines is a biotechnology company founded in 2020 and located in Cambridge, Massachusetts. The company focuses on developing small molecule drugs that target the underlying mechanisms of disease pathology, with initial therapeutic efforts directed towards conditions such as amyotrophic lateral sclerosis (ALS) and myotonic dystrophy type 1 (DM1). Faze Medicines employs advanced screening and proteomics techniques to explore biomolecular condensate interaction networks, which aids in the identification and development of novel therapeutics. Through its innovative approach, Faze aims to provide effective treatments for patients suffering from these challenging diseases.

Vedere Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on developing gene therapy products aimed at restoring functional vision for patients who have experienced vision loss due to Inherited Retinal Degenerations (IRDs) and other causes of vision impairment, both genetic and non-genetic. Unlike many ocular gene therapies that primarily slow the progression of vision loss, Vedere Bio seeks to restore lost vision, regardless of the patient's genetic background or stage of their condition. As of September 2020, Vedere Bio operates as a subsidiary of Novartis AG.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells specifically designed to target and treat solid tumors. By utilizing advanced technology, the company aims to create chimeric antigen receptor (CAR) T-cell therapies that exhibit tumor specificity, allowing them to induce cytotoxicity only when interacting with tumor cells while sparing healthy tissues. This selective targeting mitigates the safety risks commonly associated with existing CAR-T therapies, providing a promising approach to cancer treatment that enhances efficacy and reduces potential harm to noncancerous cells.

FoRx Therapeutics AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company specializes in the development of innovative therapeutics for cancer treatment, specifically targeting key molecular pathways involved in DNA replication stress. By focusing on these novel pathways, FoRx Therapeutics aims to create first-in-class compounds that offer a new approach to targeted anticancer therapies. Through its research and development efforts, the company seeks to provide effective solutions in the fight against cancer.

Immunitas Therapeutics, Inc. is focused on developing targeted therapies for cancer patients through innovative single-cell analyses. Founded in 2019 and based in Cambridge, Massachusetts, the company utilizes a single-cell sequencing platform to explore the biology of immune cells within human tumors. This approach allows Immunitas to identify novel drug targets rooted in human biology and create key biomarkers that assist in selecting patients who may benefit from their therapies. By translating laboratory findings into clinical applications, Immunitas aims to address longstanding challenges in oncology and improve treatment options for complex cancers. The company is advancing multiple programs toward early human studies, leveraging expertise in antibody discovery and engineering to develop effective treatments.

Anokion SA is a biotechnology company based in Ecublens, Switzerland, specializing in antigen-specific immune tolerance solutions. As a spin-off from the Ecole Polytechnique Fédérale de Lausanne, Anokion develops innovative immunotherapy aimed at reducing the immunogenicity of therapeutic proteins. The company's platform technology can be applied across a wide range of clinical indications, targeting both prevalent and rare autoimmune diseases, including celiac disease, multiple sclerosis, and type 1 diabetes. By engineering proteins or antigens for precise therapy, Anokion aims to provide effective treatment options that minimize side effects, thereby improving patient outcomes in the management of autoimmune and allergic diseases.

Renovacor, Inc. is a preclinical-stage biopharmaceutical company based in Philadelphia, Pennsylvania, dedicated to developing gene therapies for cardiovascular diseases. Founded in 2013, the company focuses on innovative treatments for dilated cardiomyopathy (DCM), particularly cases caused by mutations in the BAG3 gene. This condition affects over 3 million individuals in the United States, with around 35,000 patients estimated to have BAG3 mutations, classifying it as an orphan disease. Patients with BAG3-associated DCM, often younger and experiencing faster disease progression, currently have limited treatment options, primarily consisting of standard heart failure care. Renovacor's lead program involves a recombinant adeno-associated virus (AAV)-based gene therapy aimed at replacing the defective BAG3 gene, which holds the potential to prevent disease progression and improve outcomes for this vulnerable patient population.

Ayala Pharmaceuticals is a clinical-stage biopharmaceutical company based in Rehovot, Israel, founded in 2017. The company specializes in developing small molecule therapeutics for rare and aggressive cancers, focusing on genetically defined patient populations. Its lead product candidate is AL101, an intravenous gamma secretase inhibitor currently in Phase II clinical trials for treating recurrent/metastatic adenoid cystic carcinoma in patients with Notch-activating mutations. Additionally, Ayala Pharmaceuticals is developing AL102, an oral gamma secretase inhibitor in Phase I clinical trials for desmoid tumors. The company has a collaboration agreement with Novartis to develop AL102 for multiple myeloma. Ayala's approach involves using bioinformatics and next-generation sequencing to identify and address tumorigenic drivers of cancer, aiming to deliver targeted therapies to underserved patient populations.

Twentyeight-Seven Therapeutics is a developer of microRNAs designed to target regulatory proteins.The company's modulating RNA which are short non-coding RNAs that inhibit target gene expression by suppressing mRNA translation and promoting mRNA decay and develop small molecules that can modulate levels of miRNAs by targeting proteins that interact with these miRNAs, enabling users to target miRNAs are directly involved in cancer initiation, progression, and metastasis.

Poseida Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapeutics for patients with significant unmet medical needs. The company utilizes non-viral gene engineering technologies to create a pipeline of targeted therapies, including both autologous and allogeneic chimeric antigen receptor T cell (CAR-T) product candidates aimed at treating hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency, methylmalonic acidemia, and various genetic liver diseases. Founded in 2014, Poseida is committed to addressing critical health challenges through its focused research and development efforts.

Anthos Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapies for cardiovascular and metabolic diseases. Founded in 2018, the company specializes in treatments targeting Factor XI and XIa, essential components of the intrinsic coagulation pathway. Its lead product, MAA868, is an antibody designed to provide an anti-thrombotic effect, allowing healthcare professionals to prevent various thrombotic diseases while minimizing the risk of bleeding. By advancing long-acting treatment options, Anthos Therapeutics aims to improve care for patients suffering from cardiovascular conditions, representing a significant advancement over conventional therapies.

CellforCure is an innovative company specializing in advanced therapy medicinal products, particularly in the field of cell and gene therapy. It operates a cell therapy business that caters to hospitals and clinics, providing essential medicinal products designed to enhance patient care. Through its focus on advanced therapeutic solutions, CellforCure plays a significant role in the evolving landscape of medical treatments, addressing the growing demand for effective and targeted therapies.

Endocyte, Inc. is a biopharmaceutical company focused on developing receptor-targeted therapies for cancer and inflammatory diseases. Headquartered in West Lafayette, Indiana, the company creates innovative small molecule drug conjugates (SMDCs) and companion imaging agents. Among its key products, Vintafolide is in Phase IIb clinical trials for non-small cell lung cancer, while EC1456 and EC1169 are in Phase I trials for advanced solid tumors and metastatic castration-resistant prostate cancer, respectively. Additionally, Endocyte is developing several pre-clinical candidates, including EC2629 for cancer treatment and EC2319 for inflammatory diseases. The company has established collaborations with Purdue Research Foundation and other organizations to advance its pipeline. Founded in 1995, Endocyte operates as a subsidiary of Novartis AG.

Twentyeight-Seven Therapeutics is a developer of microRNAs designed to target regulatory proteins.The company's modulating RNA which are short non-coding RNAs that inhibit target gene expression by suppressing mRNA translation and promoting mRNA decay and develop small molecules that can modulate levels of miRNAs by targeting proteins that interact with these miRNAs, enabling users to target miRNAs are directly involved in cancer initiation, progression, and metastasis.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2015, the company is dedicated to creating first-in-class therapies aimed at selectively killing cancer cells. Artios's product pipeline features several promising programs, including a focus on DNA polymerase theta (Polθ), which plays a crucial role in DNA repair processes. Additionally, the company is advancing an in-licensed program that targets a newly identified protein linked to DDR and is developing a novel treatment aimed at DNA nucleases involved in these pathways. Artios collaborates with Cancer Research Technology and prominent DNA repair researchers globally to enhance its research and development efforts.

Enterprise Therapeutics, established in 2014 and headquartered in Brighton, UK, is a drug discovery company focused on developing therapies for respiratory diseases. Its primary focus is on chronic obstructive pulmonary diseases, cystic fibrosis, and asthma. The company aims to create novel treatments that target the underlying mechanisms of mucus congestion, reducing lung infections and enhancing patients' quality of life.

AveXis, now known as Novartis Gene Therapies, is a biotechnology company focused on developing and commercializing gene therapies aimed at treating rare and life-threatening neurological genetic disorders. Acquired by Novartis in 2018, the company specializes in motor neuron cell-targeted gene replacement therapies for conditions such as spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. By addressing these severe diseases, which often result in progressive muscle weakness, paralysis, and death, AveXis plays a crucial role in enhancing treatment options for affected patients and their families.

Zikani Therapeutics, Inc. is focused on developing and commercializing innovative therapeutics for patients facing limited treatment options. The company utilizes its TURBO-ZM platform to create novel ribosome modulating agents (RMAs) aimed at addressing a variety of conditions, including cystic fibrosis, familial adenomatous polyposis, APC mutant colon cancer, recessive dystrophic epidermolysis bullosa, rare cancers, and other rare genetic diseases. Additionally, Zikani is developing antibiotic compounds that target serious, multi-drug resistant gram-negative infections, an area where existing treatments are often inadequate. Founded in 2014 and initially known as Macrolide Pharmaceuticals, the company is headquartered in Watertown, Massachusetts. Through its advancements in synthetic chemistry and microbiology, Zikani Therapeutics seeks to enhance patient recovery and improve outcomes in challenging medical scenarios.

Rani Therapeutics, LLC is a clinical-stage biotherapeutics company that focuses on the development of oral delivery technologies for large drug molecules, such as peptides, proteins, and antibodies. The company has created the RaniPill capsule, a proprietary and patented platform designed to administer biologics orally, offering a convenient alternative to traditional subcutaneous or intravenous injections. This capsule is engineered to deliver precise therapeutic doses upon reaching the small intestine. Rani Therapeutics is advancing various treatments, including TNFa inhibitors for inflammatory disorders, parathyroid hormone for osteoporosis, and human growth hormone for growth deficiencies. Additionally, their portfolio includes therapies for multiple sclerosis, type II diabetes, and various inflammatory diseases. Founded in 2012 and based in San Jose, California, Rani Therapeutics has conducted several preclinical and clinical studies to assess the safety and effectiveness of its technologies, building a robust intellectual property portfolio in the process.

Aelin Therapeutics is a Belgian biotherapeutics company established in 2017 and headquartered in Heverlee. It specializes in developing innovative antibiotics and therapeutics through its proprietary Pept-in technology, which utilizes protein aggregation to achieve functional knockdown of target proteins. This platform is designed to address challenging medical conditions, including infections caused by Methicillin-resistant Staphylococcus aureus (MRSA) and other undruggable targets in humans. The company was founded by VIB in collaboration with partner universities, including KU Leuven, VUB, and UGent, and is backed by extensive preclinical proof-of-concept data. Aelin Therapeutics' approach allows for the rational design of novel biotherapeutics, distinguishing itself from traditional therapeutic modalities by targeting intracellular spaces typically inaccessible to small molecules or antibodies. The technology's versatility extends beyond human applications, demonstrating efficacy against bacteria, cancer cells, fungi, viruses, and even plant cells.

Semma Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cell therapies for patients with Type 1 diabetes. Founded in 2014, the company is dedicated to creating a transformative treatment that addresses the need for insulin injections by generating functional, insulin-producing beta cells through a proprietary method licensed from the laboratory of Professor Douglas Melton. Semma Therapeutics aims to combine these engineered cells with advanced devices to offer a potential replacement for the missing beta cells in diabetic patients, eliminating the need for immunosuppression. The company's ongoing research seeks to bring this novel therapeutic option to clinical settings, ultimately improving the lives of individuals living with diabetes.

Rani Therapeutics, LLC is a clinical-stage biotherapeutics company that focuses on the development of oral delivery technologies for large drug molecules, such as peptides, proteins, and antibodies. The company has created the RaniPill capsule, a proprietary and patented platform designed to administer biologics orally, offering a convenient alternative to traditional subcutaneous or intravenous injections. This capsule is engineered to deliver precise therapeutic doses upon reaching the small intestine. Rani Therapeutics is advancing various treatments, including TNFa inhibitors for inflammatory disorders, parathyroid hormone for osteoporosis, and human growth hormone for growth deficiencies. Additionally, their portfolio includes therapies for multiple sclerosis, type II diabetes, and various inflammatory diseases. Founded in 2012 and based in San Jose, California, Rani Therapeutics has conducted several preclinical and clinical studies to assess the safety and effectiveness of its technologies, building a robust intellectual property portfolio in the process.

Homology Medicines, Inc., established in 2015 and headquartered in Bedford, Massachusetts, is a biotechnology company focused on developing genetic medicines for patients with rare genetic diseases. The company's proprietary platform employs human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies or nuclease-free gene editing treatments in vivo, targeting various disease-relevant tissues such as the liver, central nervous system, and eye. Homology Medicines has two lead product candidates: HMI-102, currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, and HMI-103, intended for PKU treatment in pediatric patients. Additionally, the company is developing HMI-202 to treat metachromatic leukodystrophy. Homology Medicines aims to cure the underlying causes of these diseases by leveraging its robust intellectual property portfolio and experienced management team with a proven track record in rare disease therapeutics.

eFFECTOR Therapeutics Inc is a clinical-stage biopharmaceutical company based in San Diego, California. Established in 2012, the company specializes in developing selective translation regulators for cancer and other serious diseases. Its innovative approach involves small molecule drugs known as selective translation regulator inhibitors (STRIs), which target the eIF4F complex and its associated kinase, mitogen-activated protein kinase 1/2 (MNK 1/2). This complex plays a crucial role in the translation of specific messenger RNA into proteins linked to cancer progression. eFFECTOR's lead product candidate, tomivosertib, is currently being evaluated in a Phase 2b trial in combination with pembrolizumab for non-small cell lung cancer. Additionally, zotatifin, an inhibitor of eIF4A, is undergoing dose-escalation trials with plans for further expansion. The company also collaborates with Pfizer to develop inhibitors targeting eIF4E. Through these efforts, eFFECTOR aims to address the challenges of tumor growth, survival, and immune evasion in cancer treatment.

Neurovia, Inc. is a pharmaceutical company based in Cambridge, Massachusetts, founded in 2013. The company focuses on developing therapies for neurological disorders, specifically targeting unmet medical needs associated with X-linked adrenoleukodystrophy (X-ALD). Neurovia's primary objective is to provide a treatment that can halt the progression of neurological deficits caused by this rare genetic disease. The company has developed a selective thyromimetic agent aimed at improving the quality of life for patients suffering from X-ALD.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

Anaeropharma Science, Inc. is a biopharmaceutical company based in Chiyoda, Japan, founded in 2004. The company specializes in developing therapeutics aimed at treating solid cancers characterized by hypoxic environments. Utilizing its proprietary platform technology, which involves recombinant Bifidobacterium, Anaeropharma has created innovative therapies such as APS001F, an anticancer solution, and FGF2, which addresses ischemia. Its approach involves genetically enhanced bacteria that thrive in the oxygen-deprived areas of tumors, where they convert a pro-drug into a cytotoxic agent. This technology enables healthcare professionals to provide more effective and safer treatments for various cancers and related hypoxic conditions.

Vivet Therapeutics SAS is a biotechnology company focused on researching, developing, and commercializing gene therapy treatments for inherited liver disorders and orphan diseases. Founded in 2016 and based in Paris, France, the company employs a liver-targeting adeno-associated virus (AAV) vector to deliver therapeutic genes directly into hepatocytes, addressing genetic disorders at their source. Vivet is particularly known for its work on diseases such as Wilson disease, Progressive Familial Intrahepatic Cholestasis, and Citrullinemia Type I. In collaboration with the Fundacion para la Investigacion Medica Aplicada and the Centro de Investigación Medica Aplicada at the University of Navarra, Vivet aims to enhance gene delivery technologies and ensure long-term expression of therapeutic genes, thereby advancing the field of gene therapy for liver-related conditions.

Semma Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cell therapies for patients with Type 1 diabetes. Founded in 2014, the company is dedicated to creating a transformative treatment that addresses the need for insulin injections by generating functional, insulin-producing beta cells through a proprietary method licensed from the laboratory of Professor Douglas Melton. Semma Therapeutics aims to combine these engineered cells with advanced devices to offer a potential replacement for the missing beta cells in diabetic patients, eliminating the need for immunosuppression. The company's ongoing research seeks to bring this novel therapeutic option to clinical settings, ultimately improving the lives of individuals living with diabetes.

Binx Health Limited is an in vitro diagnostics company based in Trowbridge, United Kingdom, focused on developing point-of-care diagnostic solutions for infectious diseases. Established in 2005, the company offers the binx io system, a FDA-cleared and CLIA-waived platform that detects sexually transmitted infections, including chlamydia and gonorrhea, delivering results comparable to central lab performance within approximately thirty minutes. Additionally, Binx Health provides the Callisto system, which meets various throughput requirements for testing in decentralized and near-patient settings. The company's innovative approach aims to enhance access to healthcare by overcoming traditional barriers and improving patient experiences through rapid polymerase chain reaction (PCR)-based testing and a comprehensive digital program that includes counseling and follow-up care. Binx Health has also formed a strategic partnership with Sherlock Biosciences to further advance its capabilities in diagnostics.

Inflazome Ltd. is a biotechnology company based in Dublin, Ireland, founded in 2016. It specializes in developing orally available drugs aimed at addressing unmet clinical needs in inflammatory diseases by targeting the inflammasome, a component involved in the inflammatory process. The company’s innovative therapies are designed to block inflammasome signals, effectively tackling various inflammatory-driven conditions. These include orphan diseases, central nervous system disorders such as Alzheimer's and Parkinson's, systemic inflammatory diseases like cardiovascular and pulmonary conditions, and applications in ophthalmology and dermatology. By targeting the root causes of inflammation, Inflazome enables the development of precise treatments for a range of diseases, thereby enhancing therapeutic options for healthcare providers. As of September 2020, Inflazome operates as a subsidiary of Roche Holding AG.

Bionano Genomics, Inc. is a biotechnology company focused on genome mapping and analysis. It develops and markets the Saphyr system, an advanced platform designed for ultra-sensitive and specific detection of structural variations in DNA, such as insertions, deletions, and translocations, which are often linked to genetic disorders and diseases, including cancer. The Saphyr system employs optical genome mapping technology, allowing for direct visualization and analysis of long DNA molecules without relying on sequencing. This capability provides researchers and clinicians with critical insights into the structural variations within genomes, enhancing the understanding of disease mechanisms and supporting the development of personalized treatment strategies. Bionano Genomics also offers essential reagents and data solutions, including Bionano prep kits, labeling kits, and software tools for comprehensive experiment management. Its products are primarily sold to academic and governmental research laboratories, as well as pharmaceutical, biotechnology, and contract research organizations across multiple regions, including North America, Europe, and Asia. Founded in 2003 and headquartered in San Diego, California, the company was previously known as BioNano Genomics, Inc. until its name change in July 2018.

IFM Therapeutics specializes in developing small molecule medicines that target the innate immune system to address complex challenges in treating inflammatory and autoimmune disorders, as well as cancer. The company collaborates with academic partners to leverage expertise in innate immunity alongside experienced drug discovery professionals. Its subsidiary, IFM Due, focuses on discovering and developing antagonists of the cGAS/STING pathway, aiming to treat conditions such as inflammation, neuroinflammation, autoimmunity, and cancer. Additionally, IFM Discovery, a newly established incubator within the company, is engaged in advancing a portfolio of genetically validated targets for next-generation therapies addressing inflammation, neuroinflammation, autoimmunity, and fibrosis. Through these initiatives, IFM Therapeutics seeks to enhance immune response and improve the quality of life for patients facing serious health challenges.

F2G Ltd is a biotechnology company based in Manchester, United Kingdom, that specializes in developing innovative therapies for serious fungal diseases. Established in 1998, F2G focuses on its F3 series of antifungal compounds, particularly targeting challenging fungi such as Aspergillus and other filamentous molds that pose significant health risks. The company leverages its proprietary genomics technology, MycoBank, to identify critical gene targets in fungi, facilitating the creation of effective antifungal agents. Since its initial funding in 2002, F2G has expanded its research and development capabilities, enabling it to advance its patented compounds and contribute to addressing life-threatening fungal infections that affect individuals with compromised immune systems.

Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, that specializes in cellular engineering and genome editing solutions using CRISPR technology. Incorporated in 2011, the company has developed a CRISPR-Cas gene editing platform that allows precise modifications to DNA, which is applicable across various fields including human and animal therapeutics, agricultural biotechnology, and industrial biotech. Caribou's innovative approach aims to create transformative therapies, particularly in the development of allogeneic CAR-T and CAR-NK cell therapies, targeting serious diseases. The company focuses on advancing its pipeline of next-generation genome-edited cell therapies while also contributing to basic and applied biological research.

Bionano Genomics, Inc. is a biotechnology company focused on genome mapping and analysis. It develops and markets the Saphyr system, an advanced platform designed for ultra-sensitive and specific detection of structural variations in DNA, such as insertions, deletions, and translocations, which are often linked to genetic disorders and diseases, including cancer. The Saphyr system employs optical genome mapping technology, allowing for direct visualization and analysis of long DNA molecules without relying on sequencing. This capability provides researchers and clinicians with critical insights into the structural variations within genomes, enhancing the understanding of disease mechanisms and supporting the development of personalized treatment strategies. Bionano Genomics also offers essential reagents and data solutions, including Bionano prep kits, labeling kits, and software tools for comprehensive experiment management. Its products are primarily sold to academic and governmental research laboratories, as well as pharmaceutical, biotechnology, and contract research organizations across multiple regions, including North America, Europe, and Asia. Founded in 2003 and headquartered in San Diego, California, the company was previously known as BioNano Genomics, Inc. until its name change in July 2018.

Rani Therapeutics, LLC is a clinical-stage biotherapeutics company that focuses on the development of oral delivery technologies for large drug molecules, such as peptides, proteins, and antibodies. The company has created the RaniPill capsule, a proprietary and patented platform designed to administer biologics orally, offering a convenient alternative to traditional subcutaneous or intravenous injections. This capsule is engineered to deliver precise therapeutic doses upon reaching the small intestine. Rani Therapeutics is advancing various treatments, including TNFa inhibitors for inflammatory disorders, parathyroid hormone for osteoporosis, and human growth hormone for growth deficiencies. Additionally, their portfolio includes therapies for multiple sclerosis, type II diabetes, and various inflammatory diseases. Founded in 2012 and based in San Jose, California, Rani Therapeutics has conducted several preclinical and clinical studies to assess the safety and effectiveness of its technologies, building a robust intellectual property portfolio in the process.

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.

eFFECTOR Therapeutics Inc is a clinical-stage biopharmaceutical company based in San Diego, California. Established in 2012, the company specializes in developing selective translation regulators for cancer and other serious diseases. Its innovative approach involves small molecule drugs known as selective translation regulator inhibitors (STRIs), which target the eIF4F complex and its associated kinase, mitogen-activated protein kinase 1/2 (MNK 1/2). This complex plays a crucial role in the translation of specific messenger RNA into proteins linked to cancer progression. eFFECTOR's lead product candidate, tomivosertib, is currently being evaluated in a Phase 2b trial in combination with pembrolizumab for non-small cell lung cancer. Additionally, zotatifin, an inhibitor of eIF4A, is undergoing dose-escalation trials with plans for further expansion. The company also collaborates with Pfizer to develop inhibitors targeting eIF4E. Through these efforts, eFFECTOR aims to address the challenges of tumor growth, survival, and immune evasion in cancer treatment.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Spinifex is backed by a syndicate of experienced life science investors including Novo Ventures, Canaan Partners, GBS Venture Partners, Brandon Capital Partners, Uniseed and UniQuest. Founded in 2005, Spinifex raised an initial AU$3.25 million in Series A fundraising and a total of AU$23.08 in Series B funding support the further development of EMA401. Spinifex raised a US$45 million in a Series C round led by Novo Ventures and including additional new investor Canaan Partners in 2014. Spinifex has a strong portfolio of intellectual property around its lead compound EMA01, follow on candidates and the use of AT2 receptor antagonists to treat both neuropathic & inflammatory pain and restore nerve conduction velocity deficits, including granted US, European, Japanese, Chinese, Australian and New Zealand patents.

Rani Therapeutics, LLC is a clinical-stage biotherapeutics company that focuses on the development of oral delivery technologies for large drug molecules, such as peptides, proteins, and antibodies. The company has created the RaniPill capsule, a proprietary and patented platform designed to administer biologics orally, offering a convenient alternative to traditional subcutaneous or intravenous injections. This capsule is engineered to deliver precise therapeutic doses upon reaching the small intestine. Rani Therapeutics is advancing various treatments, including TNFa inhibitors for inflammatory disorders, parathyroid hormone for osteoporosis, and human growth hormone for growth deficiencies. Additionally, their portfolio includes therapies for multiple sclerosis, type II diabetes, and various inflammatory diseases. Founded in 2012 and based in San Jose, California, Rani Therapeutics has conducted several preclinical and clinical studies to assess the safety and effectiveness of its technologies, building a robust intellectual property portfolio in the process.

Merus B.V. is a clinical-stage biomedical company focused on the discovery and development of antibody-based biopharmaceuticals, specifically in the field of immuno-oncology. The company utilizes its proprietary Oligoclonics technology to produce a class of human antibodies, which allows for the creation of a mixture of therapeutic antibodies targeting a common antigen from a single cell clone. Additionally, Merus develops bispecific antibody therapeutics known as Biclonics, which are designed to engage multiple targets simultaneously. The company's pipeline includes several product candidates such as MCLA-128, MCLA-117, and MCLA-158, among others, all aimed at addressing various cancer indications.

Aduro Biotech is a clinical-stage biopharmaceutical company headquartered in Berkeley, California, established in 2000. The company specializes in the discovery, development, and commercialization of therapies that leverage the body's immune system to treat challenging diseases. Aduro is advancing several product candidates, including ADU-S100, which is undergoing various clinical trials for applications in treating advanced solid tumors, melanoma, and squamous cell carcinoma of the head and neck. Additionally, Aduro is developing BION-1301, a monoclonal antibody for IgA nephropathy, and exploring CD27, a co-stimulatory receptor, in preclinical studies. The company has formed collaboration agreements with major pharmaceutical firms such as Novartis, Eli Lilly, and Merck, alongside license agreements with research institutions like UC Berkeley and Memorial Sloan Kettering Cancer Center.

Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, that specializes in cellular engineering and genome editing solutions using CRISPR technology. Incorporated in 2011, the company has developed a CRISPR-Cas gene editing platform that allows precise modifications to DNA, which is applicable across various fields including human and animal therapeutics, agricultural biotechnology, and industrial biotech. Caribou's innovative approach aims to create transformative therapies, particularly in the development of allogeneic CAR-T and CAR-NK cell therapies, targeting serious diseases. The company focuses on advancing its pipeline of next-generation genome-edited cell therapies while also contributing to basic and applied biological research.

Aeglea BioTherapeutics, Inc. is a clinical-stage biotechnology company based in Austin, Texas, specializing in the development of human enzyme therapeutics for rare genetic and cancer-related diseases. Founded in 2013, the company is focused on addressing unmet medical needs through innovative enzyme therapies. Its lead product candidate, pegzilarginase, is a recombinant human Arginase 1 enzyme currently undergoing a Phase III trial to assess its safety and efficacy in treating Arginase 1 deficiency. Additionally, Aeglea has a preclinical pipeline that includes several candidates targeting various metabolic disorders, such as ACN00177 for homocystinuria, AEB5100 for the degradation of plasma cystine and cysteine, and AEB2109, which focuses on methionine degradation. Aeglea BioTherapeutics aims to make significant advancements in the treatment of rare metabolic diseases through its specialized enzyme therapies.

Zikani Therapeutics, Inc. is focused on developing and commercializing innovative therapeutics for patients facing limited treatment options. The company utilizes its TURBO-ZM platform to create novel ribosome modulating agents (RMAs) aimed at addressing a variety of conditions, including cystic fibrosis, familial adenomatous polyposis, APC mutant colon cancer, recessive dystrophic epidermolysis bullosa, rare cancers, and other rare genetic diseases. Additionally, Zikani is developing antibiotic compounds that target serious, multi-drug resistant gram-negative infections, an area where existing treatments are often inadequate. Founded in 2014 and initially known as Macrolide Pharmaceuticals, the company is headquartered in Watertown, Massachusetts. Through its advancements in synthetic chemistry and microbiology, Zikani Therapeutics seeks to enhance patient recovery and improve outcomes in challenging medical scenarios.

Merganser Biotech, Inc. is a research and development company focused on creating innovative medicines for the treatment of rare hematological and iron overload diseases, such as hemochromatosis, polycythemia vera, acute severe infections, and beta thalassemia. The company specializes in the development of hepcidin mimetic peptides, which are designed to regulate iron metabolism and address ineffective erythropoiesis. Founded in 2011, Merganser Biotech is headquartered in King of Prussia, Pennsylvania.

Altimmune, Inc. is a clinical stage biopharmaceutical company based in Gaithersburg, Maryland, specializing in the development of innovative treatments for liver disease, obesity, and vaccines. The company’s pipeline includes HepTcell, an immunotherapeutic candidate for chronic hepatitis B, which has successfully completed Phase I clinical trials. Altimmune also develops NasoShield, an intranasal anthrax vaccine, and NasoVAX, a recombinant intranasal vaccine. Additionally, the company is advancing AdCOVID, a single-dose intranasal vaccine for COVID-19. In the preclinical stage, Altimmune is working on ALT-801, a peptide-based dual GLP-1/glucagon receptor agonist for non-alcoholic steatohepatitis, as well as ALT-702, a tumor immunostimulant aimed at cancer treatment. The company employs proprietary technology for non-invasive intranasal delivery of its products and collaborates with institutions such as the University of Alabama at Birmingham to further its research initiatives.

Binx Health Limited is an in vitro diagnostics company based in Trowbridge, United Kingdom, focused on developing point-of-care diagnostic solutions for infectious diseases. Established in 2005, the company offers the binx io system, a FDA-cleared and CLIA-waived platform that detects sexually transmitted infections, including chlamydia and gonorrhea, delivering results comparable to central lab performance within approximately thirty minutes. Additionally, Binx Health provides the Callisto system, which meets various throughput requirements for testing in decentralized and near-patient settings. The company's innovative approach aims to enhance access to healthcare by overcoming traditional barriers and improving patient experiences through rapid polymerase chain reaction (PCR)-based testing and a comprehensive digital program that includes counseling and follow-up care. Binx Health has also formed a strategic partnership with Sherlock Biosciences to further advance its capabilities in diagnostics.

BioLineRx Ltd. is a clinical-stage biopharmaceutical development company based in Modi’in, Israel, specializing in oncology. The company's development pipeline includes several therapeutic candidates, such as BL-8040, a peptide designed for treating solid tumors, hematological malignancies, and facilitating stem cell mobilization. Another key candidate is AGI-134, an immuno-oncology agent aimed at solid tumors. Additionally, BioLineRx has developed BL-5010, a proprietary pen-like applicator intended for the non-surgical removal of skin lesions. The company has established collaborations with prominent organizations, including MSD for cancer immunotherapy, MD Anderson Cancer Center to explore the combination of BL-8040 with KEYTRUDA in pancreatic cancer, and Genentech Inc. for studies involving BL-8040 and TECENTRIQ in solid tumors. Founded in 2003, BioLineRx is focused on addressing unmet medical needs and improving existing therapies in the pharmaceutical market.

Bionano Genomics, Inc. is a biotechnology company focused on genome mapping and analysis. It develops and markets the Saphyr system, an advanced platform designed for ultra-sensitive and specific detection of structural variations in DNA, such as insertions, deletions, and translocations, which are often linked to genetic disorders and diseases, including cancer. The Saphyr system employs optical genome mapping technology, allowing for direct visualization and analysis of long DNA molecules without relying on sequencing. This capability provides researchers and clinicians with critical insights into the structural variations within genomes, enhancing the understanding of disease mechanisms and supporting the development of personalized treatment strategies. Bionano Genomics also offers essential reagents and data solutions, including Bionano prep kits, labeling kits, and software tools for comprehensive experiment management. Its products are primarily sold to academic and governmental research laboratories, as well as pharmaceutical, biotechnology, and contract research organizations across multiple regions, including North America, Europe, and Asia. Founded in 2003 and headquartered in San Diego, California, the company was previously known as BioNano Genomics, Inc. until its name change in July 2018.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

Aileron Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, dedicated to developing innovative therapeutics, particularly stabilized cell-permeating alpha-helical peptides, for oncology and other therapeutic areas. The company’s lead product candidate, ALRN-6924, is designed to treat advanced solid tumors and is currently undergoing various clinical trials, including Phase 2a studies for peripheral T-cell lymphoma and advanced solid tumors, as well as Phase 1 trials for acute myeloid leukemia and myelodysplastic syndromes. Aileron Therapeutics also explores next-generation wild type p53 reactivators and has established collaborations with notable institutions such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer to evaluate the effectiveness of ALRN-6924 in combination with other treatments. Founded in 2001 and originally known as Renegade Therapeutics, the company aims to address critical cell functions through its novel approach to modulating protein-protein interactions.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Sorbent Therapeutics, Inc. develops treatment for end stage renal disease patients undergoing dialysis. The company develops fluid retaining polymers for therapeutic uses, such as treatment of kidney diseases. Sorbent Therapeutics, Inc. was founded in 2005 and is based in Vernon Hills, Illinois.

Anokion SA is a biotechnology company based in Ecublens, Switzerland, specializing in antigen-specific immune tolerance solutions. As a spin-off from the Ecole Polytechnique Fédérale de Lausanne, Anokion develops innovative immunotherapy aimed at reducing the immunogenicity of therapeutic proteins. The company's platform technology can be applied across a wide range of clinical indications, targeting both prevalent and rare autoimmune diseases, including celiac disease, multiple sclerosis, and type 1 diabetes. By engineering proteins or antigens for precise therapy, Anokion aims to provide effective treatment options that minimize side effects, thereby improving patient outcomes in the management of autoimmune and allergic diseases.

Binx Health Limited is an in vitro diagnostics company based in Trowbridge, United Kingdom, focused on developing point-of-care diagnostic solutions for infectious diseases. Established in 2005, the company offers the binx io system, a FDA-cleared and CLIA-waived platform that detects sexually transmitted infections, including chlamydia and gonorrhea, delivering results comparable to central lab performance within approximately thirty minutes. Additionally, Binx Health provides the Callisto system, which meets various throughput requirements for testing in decentralized and near-patient settings. The company's innovative approach aims to enhance access to healthcare by overcoming traditional barriers and improving patient experiences through rapid polymerase chain reaction (PCR)-based testing and a comprehensive digital program that includes counseling and follow-up care. Binx Health has also formed a strategic partnership with Sherlock Biosciences to further advance its capabilities in diagnostics.

Alios BioPharma is focused on the discovery and development of innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company employs a range of proprietary technologies, including small molecule activators that stimulate innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein engineering of interferons. These complementary platforms enable the development of various therapeutic products targeting serious viral infections, such as chronic hepatitis B and C, HIV, respiratory viruses like pandemic influenza, and emerging viral diseases such as SARS. Alios BioPharma’s unique chemical library of nucleoside analogs and advanced virology-based screening systems further enhance its ability to create effective antiviral agents.

Aeglea BioTherapeutics, Inc. is a clinical-stage biotechnology company based in Austin, Texas, specializing in the development of human enzyme therapeutics for rare genetic and cancer-related diseases. Founded in 2013, the company is focused on addressing unmet medical needs through innovative enzyme therapies. Its lead product candidate, pegzilarginase, is a recombinant human Arginase 1 enzyme currently undergoing a Phase III trial to assess its safety and efficacy in treating Arginase 1 deficiency. Additionally, Aeglea has a preclinical pipeline that includes several candidates targeting various metabolic disorders, such as ACN00177 for homocystinuria, AEB5100 for the degradation of plasma cystine and cysteine, and AEB2109, which focuses on methionine degradation. Aeglea BioTherapeutics aims to make significant advancements in the treatment of rare metabolic diseases through its specialized enzyme therapies.

Aileron Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, dedicated to developing innovative therapeutics, particularly stabilized cell-permeating alpha-helical peptides, for oncology and other therapeutic areas. The company’s lead product candidate, ALRN-6924, is designed to treat advanced solid tumors and is currently undergoing various clinical trials, including Phase 2a studies for peripheral T-cell lymphoma and advanced solid tumors, as well as Phase 1 trials for acute myeloid leukemia and myelodysplastic syndromes. Aileron Therapeutics also explores next-generation wild type p53 reactivators and has established collaborations with notable institutions such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer to evaluate the effectiveness of ALRN-6924 in combination with other treatments. Founded in 2001 and originally known as Renegade Therapeutics, the company aims to address critical cell functions through its novel approach to modulating protein-protein interactions.

Merus B.V. is a clinical-stage biomedical company focused on the discovery and development of antibody-based biopharmaceuticals, specifically in the field of immuno-oncology. The company utilizes its proprietary Oligoclonics technology to produce a class of human antibodies, which allows for the creation of a mixture of therapeutic antibodies targeting a common antigen from a single cell clone. Additionally, Merus develops bispecific antibody therapeutics known as Biclonics, which are designed to engage multiple targets simultaneously. The company's pipeline includes several product candidates such as MCLA-128, MCLA-117, and MCLA-158, among others, all aimed at addressing various cancer indications.

Opsona Therapeutics is a drug development firm based in Dublin, Ireland, specializing in innovative approaches to immunology. Founded in 2004, the company focuses on developing novel therapeutic strategies that target the innate immune system, which plays a crucial role in various major human diseases. These include autoimmune and inflammatory diseases, cancer, transplant rejection, diabetes, Alzheimer's disease, and atherosclerosis. By modulating the human innate immune response, Opsona aims to create new drug candidates that can significantly improve treatment outcomes for patients facing these challenging health issues.

eFFECTOR Therapeutics Inc is a clinical-stage biopharmaceutical company based in San Diego, California. Established in 2012, the company specializes in developing selective translation regulators for cancer and other serious diseases. Its innovative approach involves small molecule drugs known as selective translation regulator inhibitors (STRIs), which target the eIF4F complex and its associated kinase, mitogen-activated protein kinase 1/2 (MNK 1/2). This complex plays a crucial role in the translation of specific messenger RNA into proteins linked to cancer progression. eFFECTOR's lead product candidate, tomivosertib, is currently being evaluated in a Phase 2b trial in combination with pembrolizumab for non-small cell lung cancer. Additionally, zotatifin, an inhibitor of eIF4A, is undergoing dose-escalation trials with plans for further expansion. The company also collaborates with Pfizer to develop inhibitors targeting eIF4E. Through these efforts, eFFECTOR aims to address the challenges of tumor growth, survival, and immune evasion in cancer treatment.

Opsona Therapeutics is a drug development firm based in Dublin, Ireland, specializing in innovative approaches to immunology. Founded in 2004, the company focuses on developing novel therapeutic strategies that target the innate immune system, which plays a crucial role in various major human diseases. These include autoimmune and inflammatory diseases, cancer, transplant rejection, diabetes, Alzheimer's disease, and atherosclerosis. By modulating the human innate immune response, Opsona aims to create new drug candidates that can significantly improve treatment outcomes for patients facing these challenging health issues.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Aileron Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, dedicated to developing innovative therapeutics, particularly stabilized cell-permeating alpha-helical peptides, for oncology and other therapeutic areas. The company’s lead product candidate, ALRN-6924, is designed to treat advanced solid tumors and is currently undergoing various clinical trials, including Phase 2a studies for peripheral T-cell lymphoma and advanced solid tumors, as well as Phase 1 trials for acute myeloid leukemia and myelodysplastic syndromes. Aileron Therapeutics also explores next-generation wild type p53 reactivators and has established collaborations with notable institutions such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer to evaluate the effectiveness of ALRN-6924 in combination with other treatments. Founded in 2001 and originally known as Renegade Therapeutics, the company aims to address critical cell functions through its novel approach to modulating protein-protein interactions.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

F2G Ltd is a biotechnology company based in Manchester, United Kingdom, that specializes in developing innovative therapies for serious fungal diseases. Established in 1998, F2G focuses on its F3 series of antifungal compounds, particularly targeting challenging fungi such as Aspergillus and other filamentous molds that pose significant health risks. The company leverages its proprietary genomics technology, MycoBank, to identify critical gene targets in fungi, facilitating the creation of effective antifungal agents. Since its initial funding in 2002, F2G has expanded its research and development capabilities, enabling it to advance its patented compounds and contribute to addressing life-threatening fungal infections that affect individuals with compromised immune systems.

Sorbent Therapeutics, Inc. develops treatment for end stage renal disease patients undergoing dialysis. The company develops fluid retaining polymers for therapeutic uses, such as treatment of kidney diseases. Sorbent Therapeutics, Inc. was founded in 2005 and is based in Vernon Hills, Illinois.

Celladon Corporation is a biotechnology company focused on developing molecular therapies for the treatment of heart failure. Founded in 2000 and headquartered in La Jolla, California, the company is known for its product candidates that address key enzyme deficiencies associated with advanced heart failure. One of its primary products, SERCA2a, is an enzyme that plays a crucial role in calcium cycling and contractility in heart muscle cells. Additionally, Celladon is developing MYDICAR, an enzyme replacement therapy aimed at improving heart function in patients suffering from heart failure.