SR One

Windward Bio is a clinical-stage drug development company focused on improving outcomes for individuals with advanced immunological diseases, particularly severe respiratory conditions. The company is advancing a monoclonal antibody targeting thymic stromal lymphopoietin (TSLP) into phase 2 development while creating innovative bispecific programs for various immunological disorders. Led by a team of biopharmaceutical executives, Windward Bio aims to address significant unmet medical needs in this field.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Antag Therapeutics ApS is a biotechnology company based in Copenhagen, Denmark, founded in 2017. The company specializes in developing peptide drugs aimed at treating dietary-related metabolic diseases, including obesity, diabetes, and non-alcoholic fatty liver disease. Antag Therapeutics focuses on creating peptide-based GIP receptor antagonists, which are designed to monitor and manage these metabolic conditions by reducing blood flow and lipid uptake in adipose tissue.

Avenzo Therapeutics is a clinical-stage company focused on developing innovative oncology therapies aimed at treating cancer. The company’s platform is dedicated to creating novel treatments specifically for underserved cancers, including rare and genomically defined types. By advancing a pipeline of targeted drug candidates, Avenzo Therapeutics seeks to enhance treatment options for both researchers and patients, addressing significant gaps in current cancer care.

Nomic is a bioengineering company focused on making the human proteome as accessible as the human genome. The company is developing the nELISA, a next-generation platform for measuring proteins at high throughput and scale. Nomic provides scientists with protein profiling solutions, allowing them to determine the scope and scale of their experiments. Their platform features cost-effective measurement of protein data and integrates smoothly with industry-standard cytometers for high-throughput analysis. Additionally, Nomic's technology enables the development of custom protein panels, leveraging nanotechnology to use DNA as a versatile linker to address issues of cross-reactivity in protein measurement.

Elektrofi is a preclinical-stage company based in Boston, Massachusetts, focused on advancing drug delivery methods to enhance patient experiences. Established in 2016, the company has developed a formulation system capable of processing a diverse array of therapeutic molecules, including large and complex proteins such as monoclonal antibodies, bispecifics, and fusion proteins. Its innovative technology aims to improve subcutaneous administration, allowing drug manufacturers to create breakthrough therapies that empower patients with more control over how they receive essential medications.

Asceneuron SA is a biotechnology company based in Lausanne, Switzerland, founded in 2012, that focuses on discovering and developing therapeutics for neurodegenerative disorders with significant unmet medical needs. The company targets conditions such as progressive supranuclear palsy, orphan tauopathies, Alzheimer’s disease, and Parkinson’s disease. Asceneuron aims to develop orally bioavailable small molecule drugs that address both symptomatic relief and disease progression for these debilitating conditions. Tauopathies, characterized by the accumulation of tau protein deposits in the brain, are a key area of focus, as they currently lack effective treatments. With the increasing prevalence of Alzheimer's disease posing a major healthcare challenge, Asceneuron aspires to become a leading entity in the biotech sector by providing innovative and more effective therapeutic options for patients suffering from neurodegenerative diseases. The company operates as a subsidiary of Merck KGaA.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Zenas BioPharma is a clinical-stage biopharmaceutical company based in Florida, dedicated to developing immune-based therapies for patients. The company's lead product candidate, obexelimab, is a bifunctional monoclonal antibody that targets both CD19 and FcγRIIb, which are found on B cells. This innovative approach aims to inhibit the activity of B cells involved in various autoimmune diseases while preserving their overall population, thus offering a potential therapeutic advantage over traditional treatments that deplete these cells.

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.

MISSION Therapeutics is a biotechnology company specializing in developing drugs that target the cellular process of deubiquitylation for the treatment of cancer and other diseases. Their focus is on developing small molecule drugs that selectively inhibit specific deubiquitylating enzymes (DUBs), which play crucial roles in regulating protein degradation and cellular processes. These drugs aim to intervene in disease mechanisms where DUB dysregulation is implicated, offering potential therapeutic benefits.

Alumis is a clinical-stage biopharmaceutical company dedicated to developing precision medicines for autoimmune disorders. By leveraging a proprietary data analytics platform and biological insights, Alumis aims to transform the treatment landscape for patients suffering from these conditions. The company focuses on identifying and advancing therapeutics based on genetically validated targets, which enables the creation of targeted therapies that significantly improve patient outcomes. With a team of experts experienced in drug discovery and immunology, Alumis seeks to replace broad immunosuppression with more effective, targeted treatment options, ultimately enhancing the quality of life for individuals affected by autoimmune diseases.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing therapies for idiopathic pulmonary fibrosis (IPF) and other severe respiratory diseases. The company is advancing inhaled formulations of pirfenidone, specifically AP01, which aims to enhance drug delivery to the lungs while potentially reducing side effects associated with oral treatments. This innovative approach seeks to improve the care and outcomes for patients suffering from these conditions. Established in 2011, Avalyn Pharma operates from its headquarters in Seattle, Washington, and maintains an additional office in San Diego, California. The company was previously known as Genoa Pharmaceuticals, Inc. before rebranding in July 2017.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

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.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Avenzo Therapeutics is a clinical-stage company focused on developing innovative oncology therapies aimed at treating cancer. The company’s platform is dedicated to creating novel treatments specifically for underserved cancers, including rare and genomically defined types. By advancing a pipeline of targeted drug candidates, Avenzo Therapeutics seeks to enhance treatment options for both researchers and patients, addressing significant gaps in current cancer care.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.

Rezo Therapeutics is a biotechnology company that focuses on developing precision therapeutics by mapping disease networks. Utilizing its proprietary Sequence to Systems to Drugs (SSD) platform, Rezo integrates various technologies, including proteomics, genetics, structural biology, chemistry, and bioinformatics, to create comprehensive molecular maps of diseases. This innovative approach enhances the identification of novel targets and therapies, particularly in oncology, where Rezo initially concentrates its efforts. The company aims to expand its therapeutic focus through collaborations and partnerships, positioning itself at the forefront of advancing treatment options across multiple disease areas.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

MiroBio Ltd is a biotechnology company based in Oxford, United Kingdom, focused on developing antibody modulators for immune cell receptors aimed at treating autoimmune diseases. Founded in 2018, MiroBio leverages pioneering research from the University of Oxford to understand how immune cells communicate and are activated. This foundational knowledge allows the company to create innovative antibodies that stimulate specific immune cell signals, harnessing the body's natural control mechanisms to restore balance within the immune system. By activating these natural pathways, MiroBio's therapeutics aim to provide significant benefits to patients suffering from various autoimmune conditions, enabling clinicians to effectively manage overactive immune responses associated with these diseases.

Ancora Biotech is a biotechnology company dedicated to the development of antibody biotherapeutics aimed at treating life-threatening diseases. The company specializes in clinical development support for conditions such as B cell cancers, inflammatory and metabolic disorders, and chronic hepatitis B infections. By focusing on these areas, Ancora Biotech aims to provide healthcare professionals with effective treatment options to improve patient outcomes in various critical health situations.

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.

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.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

Simcha Therapeutics is a biopharmaceutical company focused on developing engineered cytokine immunotherapies for cancer treatment. Based in New Haven, Connecticut, the company employs directed evolution to create novel cytokines aimed at enhancing the immune system's ability to combat diseases. Its lead program, ST-067, is a designer IL-18 cytokine that has demonstrated significant antitumor activity in animal studies, both as a standalone treatment and in combination with anti-PD-1 checkpoint inhibitors. Founded in 2018, Simcha Therapeutics is dedicated to advancing innovative therapies that empower patients to effectively fight cancer.

Nomic is a bioengineering company focused on making the human proteome as accessible as the human genome. The company is developing the nELISA, a next-generation platform for measuring proteins at high throughput and scale. Nomic provides scientists with protein profiling solutions, allowing them to determine the scope and scale of their experiments. Their platform features cost-effective measurement of protein data and integrates smoothly with industry-standard cytometers for high-throughput analysis. Additionally, Nomic's technology enables the development of custom protein panels, leveraging nanotechnology to use DNA as a versatile linker to address issues of cross-reactivity in protein measurement.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

HotSpot Therapeutics is a biotechnology startup based in Boston, Massachusetts, established in 2017. The company specializes in the development of allosteric therapies that target regulatory sites on proteins. Its innovative therapeutic platform is designed to identify hotspots and locate pathway treatments for cancer, metabolic disorders, and rare diseases. By leveraging a comprehensive database of hotspot structures and utilizing data science, HotSpot Therapeutics employs a multidisciplinary approach to drug discovery. This approach aims to enhance the effectiveness of treatments and improve patient outcomes across a range of conditions.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

ARS Pharmaceuticals, Inc., established in 2015 and headquartered in San Diego, California, is a biopharmaceutical company focused on developing innovative treatments for severe allergic reactions. The company has created a novel intranasal epinephrine spray known as neffy, designed for the emergency treatment of Type I allergic reactions, including anaphylaxis. Neffy utilizes a proprietary formulation that includes an absorption enhancer called Intravail, enabling it to deliver epinephrine effectively and rapidly through the nasal mucosa. This unique technology provides a convenient, reliable, and easy-to-use alternative for individuals at risk of life-threatening allergic reactions due to food, medications, or insect stings.

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.

Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.

BioTheryX, Inc. is a clinical-stage biopharmaceutical company based in Chappaqua, New York, focused on developing innovative therapies that restore protein homeostasis through targeted protein degradation. The company specializes in Protein Homeostatic Modulators and small molecule targeted protein degraders (TPDs), which utilize a unique mechanism involving molecular glues to regulate protein levels within the body. Their proprietary PRODEGY platform is built on a library of Cereblon binders, enabling the design of bifunctional and hybrid degraders aimed at treating various diseases, particularly cancer. BioTheryX's lead candidate, BTX-A51, is an oral small molecule and multi-kinase inhibitor targeting specific leukemic stem cells and oncogenic transcription factors. Established in 2007, BioTheryX aims to address significant unmet medical needs by leveraging its expertise in protein modulation and degradation.

Arcellx is a clinical-stage biotechnology company focused on developing innovative cell therapies for patients with cancer and other incurable diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Its lead product candidate, CART-ddBCMA, targets relapsed or refractory multiple myeloma and is currently undergoing a Phase 1 clinical study. This therapy has received multiple designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is advancing its controllable CAR-T therapy, ARC-SparX, through two clinical programs aimed at treating relapsed or refractory multiple myeloma and acute myeloid leukemia.

River 2 Renal is a developer of novel therapies intended to cure orphan diseases.

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.

River 3 Renal is a developer of novel therapies designed to target orphan diseases in the renal space. The company's technology has the potential to address the unmet need in several kidney diseases, by further improving the standard of care, enabling medical practitioners to treat patients in a better way. River 3 Renal was established in 2020 in New York, USA.

F-Star Therapeutics is a biopharmaceutical company focused on developing novel bispecific antibody products aimed at improving upon current standards of care, particularly in cancer therapy. The company is uniquely positioned with a strong patent portfolio, enabling it to create and develop Fcab antibody fragments and bispecific antibodies by modifying the constant region of an antibody. F-Star's Modular Antibody Technology allows for rapid discovery and development of bispecific antibodies by adding binding sites to the constant region, facilitating easier production compared to other antibody formats. The company is leveraging this technology to build a pipeline of product candidates. Founded in 2006, F-Star has received significant funding from leading venture capital firms and strategic corporate investors. The company operates out of Cambridge, UK, with over 35 employees dedicated to research and development.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

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.

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.

Palleon Pharmaceuticals is a biotechnology company that develops therapeutics targeting glycan-mediated immune regulation for the treatment of cancer and inflammatory diseases. Founded in 2015 and based in Waltham, Massachusetts, the company specializes in creating glycoimmune checkpoint inhibitors, a novel class of drugs designed to enhance immune response against cancer. By integrating insights from global leaders in glycoscience and human immunology, Palleon aims to provide a broader array of combination therapies that not only address cancer but also combat resistance to traditional immuno-oncology treatments. Additionally, the company develops therapies for inflammatory diseases, including autoimmunity and fibrosis.

Tranquis Therapeutics, Inc. is a biotechnology company focused on developing immuno-therapeutics for neurodegenerative and aging-related diseases. Established in 2016 and based in San Mateo, California, the company is rooted in neuro-immunology research from Stanford University. Its lead candidate, TQS-168, targets myeloid immune cell dysfunction associated with various nervous system disorders. The therapeutic aims to address conditions such as amyotrophic lateral sclerosis, frontotemporal dementia, Parkinson’s disease, and age-related cognitive impairment. Tranquis Therapeutics is dedicated to advancing innovative treatments that could significantly alleviate the burden of these illnesses on patients and society. The company plans to initiate development for orphan diseases like ALS and FTD before expanding to more prevalent conditions such as Parkinson's and Alzheimer's disease.

MISSION Therapeutics is a biotechnology company specializing in developing drugs that target the cellular process of deubiquitylation for the treatment of cancer and other diseases. Their focus is on developing small molecule drugs that selectively inhibit specific deubiquitylating enzymes (DUBs), which play crucial roles in regulating protein degradation and cellular processes. These drugs aim to intervene in disease mechanisms where DUB dysregulation is implicated, offering potential therapeutic benefits.

HotSpot Therapeutics is a biotechnology startup based in Boston, Massachusetts, established in 2017. The company specializes in the development of allosteric therapies that target regulatory sites on proteins. Its innovative therapeutic platform is designed to identify hotspots and locate pathway treatments for cancer, metabolic disorders, and rare diseases. By leveraging a comprehensive database of hotspot structures and utilizing data science, HotSpot Therapeutics employs a multidisciplinary approach to drug discovery. This approach aims to enhance the effectiveness of treatments and improve patient outcomes across a range of conditions.

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.

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.

MiroBio Ltd is a biotechnology company based in Oxford, United Kingdom, focused on developing antibody modulators for immune cell receptors aimed at treating autoimmune diseases. Founded in 2018, MiroBio leverages pioneering research from the University of Oxford to understand how immune cells communicate and are activated. This foundational knowledge allows the company to create innovative antibodies that stimulate specific immune cell signals, harnessing the body's natural control mechanisms to restore balance within the immune system. By activating these natural pathways, MiroBio's therapeutics aim to provide significant benefits to patients suffering from various autoimmune conditions, enabling clinicians to effectively manage overactive immune responses associated with these diseases.

Arcellx is a clinical-stage biotechnology company focused on developing innovative cell therapies for patients with cancer and other incurable diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Its lead product candidate, CART-ddBCMA, targets relapsed or refractory multiple myeloma and is currently undergoing a Phase 1 clinical study. This therapy has received multiple designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is advancing its controllable CAR-T therapy, ARC-SparX, through two clinical programs aimed at treating relapsed or refractory multiple myeloma and acute myeloid leukemia.

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.

Turning Point Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative small molecule targeted therapies for oncology. The company designs a range of tyrosine kinase inhibitors (TKIs) aimed at addressing genetic drivers of cancer in both TKI-naïve and TKI-pretreated patients. Its lead drug candidate, repotrectinib, is currently under evaluation in the ongoing Phase I/II TRIDENT-1 trial, targeting patients with ROS1+ advanced non-small-cell lung cancer and those with ROS1+, NTRK+, or ALK+ advanced solid tumors. Additionally, Turning Point's pipeline includes other multi-targeted drug candidates, such as TPX-0046, a RET/SRC inhibitor; TPX-0022, a MET/CSF1R/SRC inhibitor; and TPX-0131, which is an ALK inhibitor in preclinical development. Founded in 2013 and headquartered in San Diego, California, the company aims to advance precision medicine in oncology by addressing unmet medical needs.

Gotham Therapeutics is a biotechnology company based in New York that focuses on developing a novel class of drugs targeting epitranscriptomics machinery. Founded in 2017, the company aims to provide new treatment options for patients with cancers, autoimmune disorders, and neurodegenerative diseases. Its approach involves creating small molecules that modify the activity of messenger ribonucleic acid (mRNA) to influence protein transcription. This innovative strategy allows Gotham Therapeutics to potentially optimize therapeutic outcomes for individuals suffering from serious health conditions.

Morphic Therapeutic is a biotechnology company focused on developing oral integrin therapies aimed at treating serious chronic diseases such as autoimmune disorders, cardiovascular conditions, metabolic diseases, fibrosis, and cancer. The company specializes in creating small molecule inhibitors that target various integrins. By leveraging insights into integrin biology, Morphic Therapeutic is building a pipeline of innovative therapeutics designed to address a range of immunological, fibrotic, neoplastic, and vascular diseases, thereby offering potential new treatment options for patients with significant unmet medical needs.

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.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Propeller Health is a digital health platform focused on improving the management of chronic respiratory diseases such as asthma and COPD. The company provides a mobile application and sensors that attach to inhalers, enabling users to track their medication usage, triggers, and symptoms. This data is stored securely in a cloud-based infrastructure and facilitates personalized feedback and education for users. The platform also allows users to set medication reminders and alerts, as well as share their health data with physicians and family members. Propeller Health collaborates with various healthcare organizations and has received multiple awards for innovation in the field. Founded by David Van Sickle, Greg Tracy, and Mark Gehring, the company operates from Wisconsin, United States.

Constellation Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for cancers linked to abnormal gene expression and drug resistance. The company specializes in the field of epigenetics, targeting selective regulators of epigenetic function that are vital for gene expression control. Its lead product candidates include CPI-0610, currently in Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is undergoing Phase Ib/II clinical trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is advancing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial aimed at treating solid tumors. Founded in 2008 and based in Cambridge, Massachusetts, the company was previously known as Epigenetix, Inc. before rebranding in March 2008.

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.

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.

Atox Bio Inc. is a biotechnology company based in Ness Ziona, Israel, with an additional office in North Carolina. Founded in 2003, the company specializes in developing immunomodulators aimed at treating critically ill patients suffering from severe infections and acute inflammatory conditions. Atox Bio focuses on novel therapies that target multiple pathways within both the adaptive and innate immune responses, which are crucial in managing infectious diseases, inflammatory disorders, and ischemia/reperfusion injuries. The company’s products are designed to address serious medical conditions with high morbidity and mortality, showing promising results in improving patient outcomes and reducing tissue damage during acute inflammatory events.

Palleon Pharmaceuticals is a biotechnology company that develops therapeutics targeting glycan-mediated immune regulation for the treatment of cancer and inflammatory diseases. Founded in 2015 and based in Waltham, Massachusetts, the company specializes in creating glycoimmune checkpoint inhibitors, a novel class of drugs designed to enhance immune response against cancer. By integrating insights from global leaders in glycoscience and human immunology, Palleon aims to provide a broader array of combination therapies that not only address cancer but also combat resistance to traditional immuno-oncology treatments. Additionally, the company develops therapies for inflammatory diseases, including autoimmunity and fibrosis.

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.

Turning Point Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative small molecule targeted therapies for oncology. The company designs a range of tyrosine kinase inhibitors (TKIs) aimed at addressing genetic drivers of cancer in both TKI-naïve and TKI-pretreated patients. Its lead drug candidate, repotrectinib, is currently under evaluation in the ongoing Phase I/II TRIDENT-1 trial, targeting patients with ROS1+ advanced non-small-cell lung cancer and those with ROS1+, NTRK+, or ALK+ advanced solid tumors. Additionally, Turning Point's pipeline includes other multi-targeted drug candidates, such as TPX-0046, a RET/SRC inhibitor; TPX-0022, a MET/CSF1R/SRC inhibitor; and TPX-0131, which is an ALK inhibitor in preclinical development. Founded in 2013 and headquartered in San Diego, California, the company aims to advance precision medicine in oncology by addressing unmet medical needs.

Progyny, Inc. is a benefits management company focused on providing comprehensive fertility and family building benefits solutions for employers in the United States. Established in 2008 and headquartered in New York City, Progyny offers a range of services including a differentiated benefits plan design, personalized support through Patient Care Advocates, and access to a selective network of high-quality fertility specialists. The company's fertility benefits solution encompasses treatment services known as Smart Cycles, as well as an integrated pharmacy benefits solution called Progyny Rx, which facilitates access to necessary medications during treatment. Additionally, Progyny addresses the financial aspects of family building by covering surrogacy and adoption expenses for its clients and their employees. Formerly known as Auxogyn, Inc., the company rebranded to Progyny, Inc. in 2015.

ZappRx, Inc. is a digital health company based in Boston, Massachusetts, that specializes in a cloud-based platform designed to streamline the management of specialty medications. Founded in 2012, the company provides a secure online environment where healthcare providers, specialty pharmacies, and biopharma companies can collaborate efficiently. Its platform automates the prescription and prior authorization processes, facilitating communication among patients, physicians, and pharmacists while reducing reliance on traditional methods such as faxes and phone calls. By consolidating essential information related to specialty prescribing—including pharmacy details, payer requirements, and clinical history—the platform enhances the efficiency and transparency of healthcare delivery, particularly in the fields of pulmonary, cardiology, and gastroenterology. ZappRx operates as a subsidiary of Allscripts Healthcare Solutions, Inc. as of June 2019.

Pulmocide Ltd is a London-based company established in 2007 that specializes in developing inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. The company focuses on creating a new generation of antifungal drugs tailored for inhalation, which enhances the delivery of medication directly to the lungs while reducing systemic exposure. This targeted approach is designed to improve efficacy against respiratory aspergillosis and minimize systemic toxicities, thereby offering safe and effective treatment options for patients with acute and chronic respiratory diseases.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Propeller Health is a digital health platform focused on improving the management of chronic respiratory diseases such as asthma and COPD. The company provides a mobile application and sensors that attach to inhalers, enabling users to track their medication usage, triggers, and symptoms. This data is stored securely in a cloud-based infrastructure and facilitates personalized feedback and education for users. The platform also allows users to set medication reminders and alerts, as well as share their health data with physicians and family members. Propeller Health collaborates with various healthcare organizations and has received multiple awards for innovation in the field. Founded by David Van Sickle, Greg Tracy, and Mark Gehring, the company operates from Wisconsin, United States.

True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.

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.

Arch Oncology, Inc. is an immuno-oncology company focused on discovering and developing antibody therapeutics for the treatment of solid tumors and hematologic cancers. Established in 2006 and headquartered in Brisbane, California, with a research laboratory in St. Louis, Missouri, the company specializes in anti-CD47 antibodies that represent a novel class of checkpoint inhibitors. These second-generation antibodies are designed to engage both the adaptive and innate immune systems, effectively targeting and killing tumor cells. By harnessing the body's immune response, Arch Oncology aims to provide new treatment options for patients battling cancer.

Progyny, Inc. is a benefits management company focused on providing comprehensive fertility and family building benefits solutions for employers in the United States. Established in 2008 and headquartered in New York City, Progyny offers a range of services including a differentiated benefits plan design, personalized support through Patient Care Advocates, and access to a selective network of high-quality fertility specialists. The company's fertility benefits solution encompasses treatment services known as Smart Cycles, as well as an integrated pharmacy benefits solution called Progyny Rx, which facilitates access to necessary medications during treatment. Additionally, Progyny addresses the financial aspects of family building by covering surrogacy and adoption expenses for its clients and their employees. Formerly known as Auxogyn, Inc., the company rebranded to Progyny, Inc. in 2015.

Morphic Therapeutic is a biotechnology company focused on developing oral integrin therapies aimed at treating serious chronic diseases such as autoimmune disorders, cardiovascular conditions, metabolic diseases, fibrosis, and cancer. The company specializes in creating small molecule inhibitors that target various integrins. By leveraging insights into integrin biology, Morphic Therapeutic is building a pipeline of innovative therapeutics designed to address a range of immunological, fibrotic, neoplastic, and vascular diseases, thereby offering potential new treatment options for patients with significant unmet medical needs.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

MISSION Therapeutics is a biotechnology company specializing in developing drugs that target the cellular process of deubiquitylation for the treatment of cancer and other diseases. Their focus is on developing small molecule drugs that selectively inhibit specific deubiquitylating enzymes (DUBs), which play crucial roles in regulating protein degradation and cellular processes. These drugs aim to intervene in disease mechanisms where DUB dysregulation is implicated, offering potential therapeutic benefits.

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.

Navitor Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing small molecule drugs that target mTORC1, a key regulator of cellular metabolism and renewal, to treat age-related diseases. Utilizing a proprietary platform, Navitor's therapies can selectively inhibit or activate mTORC1, which plays a crucial role in cellular processes. One of its notable products, NV-5138, is an orally bioavailable small molecule designed to activate mTORC1, particularly addressing its suppression in the brain associated with depression. Additionally, the company's N-Valog program focuses on selective inhibition of mTORC1 for chronic kidney disease, with implications for other age-related conditions. Founded in 2009 and headquartered in Cambridge, Massachusetts, Navitor Pharmaceuticals aims to unlock the therapeutic potential of the mTOR pathway by modulating nutrient-sensing proteins, thereby addressing various metabolic, neurodegenerative, autoimmune, and musculoskeletal diseases.

True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.

Puridify Limited is a bioprocessing company based in Stevenage, United Kingdom, established in 2013. It specializes in the development of innovative purification technologies aimed at enhancing the manufacturing of industrial biomolecules. The company has created FibroSelect, a nanofiber-based platform purification technology that addresses limitations associated with traditional packed bed chromatography systems and chromatography membranes. This technology is designed to provide consistent purification performance across various scales, making it particularly suitable for the purification of macromolecular biotherapeutics such as gene therapies, viral vectors, and fusion proteins. By improving purification efficiencies, Puridify enables the production of cost-effective biomolecule products, supporting advancements in the biopharmaceutical sector. As of late 2017, Puridify operates as a subsidiary of GE Healthcare Limited.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

Asceneuron SA is a biotechnology company based in Lausanne, Switzerland, founded in 2012, that focuses on discovering and developing therapeutics for neurodegenerative disorders with significant unmet medical needs. The company targets conditions such as progressive supranuclear palsy, orphan tauopathies, Alzheimer’s disease, and Parkinson’s disease. Asceneuron aims to develop orally bioavailable small molecule drugs that address both symptomatic relief and disease progression for these debilitating conditions. Tauopathies, characterized by the accumulation of tau protein deposits in the brain, are a key area of focus, as they currently lack effective treatments. With the increasing prevalence of Alzheimer's disease posing a major healthcare challenge, Asceneuron aspires to become a leading entity in the biotech sector by providing innovative and more effective therapeutic options for patients suffering from neurodegenerative diseases. The company operates as a subsidiary of Merck KGaA.

Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Gladius Pharmaceuticals Inc. is a biotechnology company based in Montréal that focuses on developing innovative antibiotics to combat life-threatening, multi-drug-resistant bacterial infections. The firm is dedicated to creating novel drug candidates that offer broad-spectrum efficacy against gram-negative pathogens. Its antibiotics are designed to assist in the empiric treatment of these infections and have the potential to replace older generation cephalosporins, thereby enabling healthcare providers to treat bacterial infections more effectively.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Akamis Bio is a clinical-stage oncology company dedicated to advancing cancer treatment through its innovative Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform. The company develops a portfolio of therapeutics specifically targeting solid tumors, aiming to enhance patients' immune responses to recognize and eradicate cancer cells. By harnessing a proprietary intravenously administered viral platform, Akamis Bio focuses on delivering effective gene therapies that address the needs of cancer patients, striving to improve their quality of life and treatment outcomes.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.

ZappRx, Inc. is a digital health company based in Boston, Massachusetts, that specializes in a cloud-based platform designed to streamline the management of specialty medications. Founded in 2012, the company provides a secure online environment where healthcare providers, specialty pharmacies, and biopharma companies can collaborate efficiently. Its platform automates the prescription and prior authorization processes, facilitating communication among patients, physicians, and pharmacists while reducing reliance on traditional methods such as faxes and phone calls. By consolidating essential information related to specialty prescribing—including pharmacy details, payer requirements, and clinical history—the platform enhances the efficiency and transparency of healthcare delivery, particularly in the fields of pulmonary, cardiology, and gastroenterology. ZappRx operates as a subsidiary of Allscripts Healthcare Solutions, Inc. as of June 2019.

Thrasos is a bio-therapeutics company dedicated to discovering and developing targeted therapies for severe organ failure, with a primary emphasis on kidney diseases. The company's lead program, THR-184, aims to prevent and treat acute kidney injury (AKI). Additionally, Thrasos is advancing a preclinical development program that addresses chronic kidney disease (CKD). The company also focuses on developing innovative treatments for rare kidney-related conditions, such as myeloma cast nephropathy, which occurs when protein aggregates obstruct renal tubules, leading to the risk of permanent kidney damage. Through its efforts, Thrasos seeks to provide timely and effective treatment options for patients suffering from these serious kidney issues.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

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.