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 headquartered in Watertown, Massachusetts, that specializes in developing therapies for diseases affecting mucosal tissues using its proprietary nanoparticle-based Mucus Penetrating Particles (MPP) technology. This innovative approach enhances drug distribution and pharmacokinetics by delivering biocompatible, drug-loaded particles that uniformly coat mucosal surfaces. The company's lead product candidate, KPI-121 0.25%, has successfully completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS, another product, has also completed two Phase III trials for treating inflammation and pain following ocular surgery. Kala is advancing its pipeline, including KPI-285, a receptor tyrosine kinase inhibitor for retinal diseases, which is currently in preclinical studies. The company aims to address significant clinical needs while exploring research collaborations to enhance its product offerings.
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 develops effective therapeutics for orphan tauopathies and Alzheimer’s disease. By focusing on areas of high unmet medical need, Asceneuron aspires to become a leading biotech company specialized in small molecule drug discovery for neurodegenerative diseases. Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as e.g. neurofibrillary tangles in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major contributor to neurodegeneration and mutations in the tau gene are causative of the tauopathy fronto-temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17). Due to increasing life expectancy, Alzheimer’s disease is viewed as one of the largest healthcare problems of this century, imposing a major economic burden on societies in the Western and developing world. Current treatment options provide limited benefits supporting the urgent need for more efficacious and better tolerated medicines that address symptomatic relief as well as disease progression.
Kala Pharmaceuticals, Inc. is a biopharmaceutical company headquartered in Watertown, Massachusetts, that specializes in developing therapies for diseases affecting mucosal tissues using its proprietary nanoparticle-based Mucus Penetrating Particles (MPP) technology. This innovative approach enhances drug distribution and pharmacokinetics by delivering biocompatible, drug-loaded particles that uniformly coat mucosal surfaces. The company's lead product candidate, KPI-121 0.25%, has successfully completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS, another product, has also completed two Phase III trials for treating inflammation and pain following ocular surgery. Kala is advancing its pipeline, including KPI-285, a receptor tyrosine kinase inhibitor for retinal diseases, which is currently in preclinical studies. The company aims to address significant clinical needs while exploring research collaborations to enhance its product offerings.
Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.
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.
CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.
Odyssey Therapeutics is a biotechnology company focused on developing next-generation immunomodulators and oncology drugs. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, identifying promising targets while reducing the likelihood of failure during development. Odyssey Therapeutics is committed to advancing the field of drug discovery and expediting clinical development, ultimately contributing to the creation of more effective precision medicines for the healthcare industry.
Avalyn Pharma is a biopharma company that advances therapies for the treatment of Idiopathic Pulmonary Fibrosis (IPF) and other severe respiratory diseases. Its therapies rapidly advance AP01 for the treatment of IPF, provides additional pipeline candidates for IPF, improve the care and outcomes of severe respiratory diseases, and develop novel inhaled therapeutics. Avalyn Pharma was established in 2011 and is headquartered in Seattle, Washington.
Private Equity Round in 2023
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, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.
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's platform is based on ground-breaking research by their scientific founders at the University of Oxford delineating how immune cells communicate and how they are activated. These insights have enabled MiroBio to create antibodies that can leverage natural signalling mechanisms to restore immune system balance and control.
Developer of antibody biotherapeutics drugs intended to develop a treatment for several life-threatening diseases. The company focuses on clinical development support for the treatment of B cell cancers, inflammatory and metabolic disorders, and chronic hepatitis B infections, enabling healthcare workers to treat patients with various life-threatening 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.
Mineralys Therapeutics is a biopharmaceutical company that provides hypertension treatment. They also bring a targeted approach to the management of hypertension.
Odyssey Therapeutics is a biotechnology company focused on developing next-generation immunomodulators and oncology drugs. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, identifying promising targets while reducing the likelihood of failure during development. Odyssey Therapeutics is committed to advancing the field of drug discovery and expediting clinical development, ultimately contributing to the creation of more effective precision medicines for the healthcare industry.
Simcha Therapeutics is a biopharmaceutical company developing engineered cytokine immunotherapy for cancer. The company uses directed evolution to engineer novel cytokines designed to unlock the precision and power of the immune system. Simcha’s lead program, ST-067, is a designer IL-18 cytokine that has shown potent antitumor effects in animal models, both as a monotherapy and when combined with anti-PD-1 checkpoint inhibitors, as described in Nature in June 2020. It was founded in 2018 and is headquartered in New Haven, Connecticut, USA.
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 developing next-generation immunomodulators and oncology drugs. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, identifying promising targets while reducing the likelihood of failure during development. Odyssey Therapeutics is committed to advancing the field of drug discovery and expediting clinical development, ultimately contributing to the creation of more effective precision medicines for the healthcare industry.
HotSpot Therapeutics is a biotech startup established in 2017 and based in Boston, Massachusetts. The company specializes in developing allosteric therapies that target regulatory sites on proteins. Its innovative therapeutic platform is designed to identify hotspots and locate pathway treatments for a range of conditions, including cancer, metabolic disorders, and rare diseases. By leveraging a comprehensive database of hotspot structures and employing data science, HotSpot Therapeutics aims to enhance drug discovery and provide effective treatment options for patients with various health issues.
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 is a stealth pharmaceutical company that developed a novel intranasal epinephrine spray with a unique absorption technology that could be easy-to-use, convenient, and more reliable for patients and loved ones at risk of severe allergic reactions to food, medications and insect bites that could lead to life-threatening anaphylaxis.
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, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.
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.
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
Series A in 2021
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.
River 2 Renal
Series A in 2021
River 2 Renal is a developer of novel therapies intended to cure orphan diseases.
F-Star Therapeutics is a clinical-stage biopharmaceutical company focused on creating innovative bispecific antibody therapies aimed at improving cancer treatment. Utilizing its proprietary Modular Antibody Technology, F-Star develops bispecific antibodies that enhance the capabilities of traditional monoclonal antibodies while minimizing production challenges. The company's unique approach allows for the rapid discovery and development of these therapies, which seek to block tumor immune evasion and provide patients with more effective and durable treatment options compared to existing immuno-oncology therapies. Founded in 2006, F-Star has established a solid patent position and has attracted significant investment from leading venture capital firms and strategic corporate partners. The company has also formed alliances with major pharmaceutical companies, allowing it to expand its research and development efforts. With a dedicated team based in Cambridge, UK, F-Star is advancing a pipeline of product candidates that leverage its innovative technology.
Decibel Therapeutics is a developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.
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 biotech startup established in 2017 and based in Boston, Massachusetts. The company specializes in developing allosteric therapies that target regulatory sites on proteins. Its innovative therapeutic platform is designed to identify hotspots and locate pathway treatments for a range of conditions, including cancer, metabolic disorders, and rare diseases. By leveraging a comprehensive database of hotspot structures and employing data science, HotSpot Therapeutics aims to enhance drug discovery and provide effective treatment options for patients with various health issues.
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's platform is based on ground-breaking research by their scientific founders at the University of Oxford delineating how immune cells communicate and how they are activated. These insights have enabled MiroBio to create antibodies that can leverage natural signalling mechanisms to restore immune system balance and control.
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 developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.
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 mobile platform that offers sensors, mobile apps, analytics, and services to support respiratory health management. The HIPAA compliant platform stores users’ data in a cloud computing infrastructure. The Propeller sensor and mobile application help users learn more about and better manage their asthma, COPD, and respiratory disease. The Propeller sensor attaches to users’ inhalers and wirelessly syncs with their smartphones. It tracks their triggers and symptoms and sends personalized feedback and education to their phones based on the symptoms. Users can see how often they use the medication, and set up medication reminders and alerts. Propeller community enables users to share their Propeller data with their physician and family members. Propeller Health is backed by Safeguard Scientifics, The Social+Capital Partnership, California HealthCare Foundation, and other investors. The platform works with organizations such as Dignity Health, Wyckoff Heights Medical Center, Amerigroup Florida, and the City of Louisville. The system has been featured in The Economist, Washington Post, Fast Company, Wired, and Scientific American. Propeller Health has received TEDMED Innovation Showcase, White House Champion of Change, and Bluetooth Breakthrough Product awards. Propeller Health was launched by David Van Sickle, Greg Tracy, and Mark Gehring and its operations are based in 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 based in New York that focuses on providing fertility and family-building benefits for employers in the United States. The company offers a comprehensive fertility benefits solution that includes a differentiated plan design, personalized support through dedicated Patient Care Advocates, and access to a selective network of high-quality fertility specialists. Progyny also features Progyny Rx, an integrated pharmacy benefit that supplies necessary medications for treatment. Additionally, the company covers surrogacy and adoption expenses for its clients and their employees. Established in 2008 and rebranded from Auxogyn, Inc. in 2015, Progyny aims to create inclusive solutions that enhance patient care while reducing healthcare costs for employers.
ZappRx is a digital health company that is modernizing and simplifying the way specialty drugs are managed. We provide a secure, collaborative online platform that allows patients, physicians, and pharmacists to work together to improve patients’ access to treatment and deliver medicine faster.
Pulmocide Ltd, a company focused on the discovery and development of a new generation of inhaled medicines for the treatment of serious viral and fungal infections of the respiratory tract.
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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.
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 based 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 to treat ornithine transcarbamylase deficiency. Translate Bio aims to create innovative medicines that have the potential to significantly impact the treatment landscape for rare diseases.
Propeller Health is a mobile platform that offers sensors, mobile apps, analytics, and services to support respiratory health management. The HIPAA compliant platform stores users’ data in a cloud computing infrastructure. The Propeller sensor and mobile application help users learn more about and better manage their asthma, COPD, and respiratory disease. The Propeller sensor attaches to users’ inhalers and wirelessly syncs with their smartphones. It tracks their triggers and symptoms and sends personalized feedback and education to their phones based on the symptoms. Users can see how often they use the medication, and set up medication reminders and alerts. Propeller community enables users to share their Propeller data with their physician and family members. Propeller Health is backed by Safeguard Scientifics, The Social+Capital Partnership, California HealthCare Foundation, and other investors. The platform works with organizations such as Dignity Health, Wyckoff Heights Medical Center, Amerigroup Florida, and the City of Louisville. The system has been featured in The Economist, Washington Post, Fast Company, Wired, and Scientific American. Propeller Health has received TEDMED Innovation Showcase, White House Champion of Change, and Bluetooth Breakthrough Product awards. Propeller Health was launched by David Van Sickle, Greg Tracy, and Mark Gehring and its operations are based in Wisconsin, United States.
True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
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 based in New York that focuses on providing fertility and family-building benefits for employers in the United States. The company offers a comprehensive fertility benefits solution that includes a differentiated plan design, personalized support through dedicated Patient Care Advocates, and access to a selective network of high-quality fertility specialists. Progyny also features Progyny Rx, an integrated pharmacy benefit that supplies necessary medications for treatment. Additionally, the company covers surrogacy and adoption expenses for its clients and their employees. Established in 2008 and rebranded from Auxogyn, Inc. in 2015, Progyny aims to create inclusive solutions that enhance patient care while reducing healthcare costs for employers.
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.
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.
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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.
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 is a developer of novel medicines designed to target specific nutrient-sensing proteins that selectively regulate mTORC1 activity to treat diseases. The company's medicines selectively modulate the activity of mTORC1, a master regulator of cell growth and metabolism, and unlock the therapeutic potential of the mTOR pathway by targeting the amino acid and other nutrient sensors that selectively regulate the activity of mTORC1, enabling healthcare practitioners to address a wide range of metabolic, neurodegenerative, autoimmune and musculoskeletal diseases. It was founded in 2009 and headquartered in Cambridge, Massachusetts.
True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
Puridify conducts research and development activities on bioprocessing technologies aimed at advancing industrial biomolecule manufacture. The company was founded by Tom Haywood, Oliver Hardick, and Iwan Roberts in March 2013 and is based in United Kingdom.
Decibel Therapeutics is a developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.
Asceneuron develops effective therapeutics for orphan tauopathies and Alzheimer’s disease. By focusing on areas of high unmet medical need, Asceneuron aspires to become a leading biotech company specialized in small molecule drug discovery for neurodegenerative diseases. Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as e.g. neurofibrillary tangles in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major contributor to neurodegeneration and mutations in the tau gene are causative of the tauopathy fronto-temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17). Due to increasing life expectancy, Alzheimer’s disease is viewed as one of the largest healthcare problems of this century, imposing a major economic burden on societies in the Western and developing world. Current treatment options provide limited benefits supporting the urgent need for more efficacious and better tolerated medicines that address symptomatic relief as well as disease progression.
Principia Biopharma is a private, pre-clinical stage company initially focused on the discovery and development of differentiated small molecule drugs targeting inflammatory/autoimmune diseases as well as cancer. The company expects to submit an IND for its lead program in 2013 and continues to invest in additional programs and its reversible covalent platform.
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 based 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 to treat ornithine transcarbamylase deficiency. Translate Bio aims to create innovative medicines that have the potential to significantly impact the treatment landscape for rare diseases.
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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.
Akamis Bio is a clinical-stage oncology company dedicated to improving the lives of cancer patients through its innovative Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform. The company is focused on developing a range of therapeutics aimed at solid tumors, utilizing its proprietary T-SIGn virus platform administered intravenously. This approach aims to enhance the ability of a patient's immune system to identify, attack, and eliminate cancer cells, providing a targeted treatment option for those affected by cancer.
CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.
CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.
True North Therapeutics is a pioneering biotechnology company selectively inhibiting the Complement pathway for diseases of high unmet clinical need. The company’s lead monoclonal antibody, TNT009, targets the Classical Complement pathway, enabling selective inhibition of downstream phagocytosis, inflammation, and cell lysis. True North is currently focused on Complement-mediated rare diseases in the hematologic, kidney transplant, dermatology, and neurological space. True North was formed in 2013 as a spin-out of iPierian.
ZappRx is a digital health company that is modernizing and simplifying the way specialty drugs are managed. We provide a secure, collaborative online platform that allows patients, physicians, and pharmacists to work together to improve patients’ access to treatment and deliver medicine faster.
Thrasos is a bio-therapeutics company focused on the discovery and development of targeted therapies for the prevention and treatment of severe organ failure, with a principle focus on kidney disease. The Company's lead therapeutic program, THR-184, is focused on the prevention and treatment of acute kidney injury (AKI). The Company also has a preclinical development program for treating chronic kidney disease (CKD).
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.