New Enterprise Associates

New Enterprise Associates, Inc. is a private equity and venture capital firm founded in 1977 and headquartered in Menlo Park, California, with additional offices in India and China. The firm specializes in investing across various stages of business development, from seed funding to public offerings, with a strong emphasis on the technology and healthcare sectors. Its investment strategy encompasses a wide range of industries, including information technology, consumer internet, financial technology, software, healthcare services, life sciences, biopharmaceuticals, and energy technology. New Enterprise Associates actively engages with entrepreneurs to support the growth of their companies, making investments between $0.05 million and $20 million. The firm has a global investment focus, particularly in regions such as Asia, Brazil, and the United States.

Michael Albert

Associate

Kate Barrett

Partner, Communications

Peter Barris

Chairman

Forest Baskett

General Partner

Ron Bernal

Venture Partner

Zak Burns

Associate

James Buxton MD

Principal

Carmen Chang

Partner, Chair and Head of Asia

Tak Cheung

Partner

Philip Chopin

Managing Director, NEA UK

Pamela Clark

General Partner

Vanessa Deng

Associate

Chris Doppman

Principal, LP Relations

Tony Florence

General Partner

Jay Graf

Venture Partner

Lily Huang

Principal, Healthcare VC

Arjun Jain

Senior Associate

James Kaplan

Associate

Patrick Kerins

General Partner

Nick Kline

Principal, LP Relations

Vanessa Larco

Partner

Danielle Lay

Principal

Tiffany Le

Senior Associate

Michael Li

Principal

Tiffany Luck

Partner

Mohamad Makhzoumi

General Partner

Matthew McAviney

Partner

Amit Mukherjee

Partner

Bibhash Mukhopadhyay

Principal

Mike O'Dell

Venture Partner

Kemi Odusan

Senior Associate

Greg Papadopoulos

Venture Partner

Luke Pappas

Principal

Michele Park

Partner

Eileen Qian

Associate

Scott Sandell

Managing General Partner

Tim Schaller

CFO

Andrew Schoen

Partner

Jordan Shapiro

Senior Associate

Alex Sharata

Associate

Peter Sonsini

General Partner

George Stamas

Venture Partner

Brad Thawley

Partner, Limited Partner Relations

Lila Tretikov

Partner

Paul Walker

General Partner

Blake Wu

Partner

Lulu Xu

Principal

Rick Yang

Partner, Head of Technology

Emily Zhen

Associate

Ann Bordetsky

Partner

Arno Penzias Ph.D

Venture Partner

J.C. Lopez

Principal

Past deals in Therapeutics

Fire1

Venture Round in 2025
FIRE1 and is based in Dublin, Ireland, will focus on developing novel therapeutic devices.

Pathos

Series C in 2024
Pathos is a company that focuses on enhancing the drug discovery process through its AI-enabled platform. This self-learning technology leverages big data and artificial intelligence to streamline the identification and validation of drug targets, ultimately establishing their clinical efficacy. By facilitating the exploration of novel concepts, Pathos aims to support drug developers in advancing innovative therapies. The platform is designed to manage the entire end-to-end process of designing and developing new therapeutics, thereby significantly reducing the time required for medication development.

Amber Therapeutics

Series A in 2024
Amber Therapeutics develops the next-generation of bioelectrical therapies that can sense and adapt as well as stimulate, with the potential to radically improve clinical outcomes in patients with functional disorders of the peripheral nervous system.

Zenas BioPharma

Series C in 2024
Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.

Xaira Therapeutics

Venture Round in 2024
Xaira Therapeutics is an integrated biotechnology company focused on revolutionizing drug discovery and development through the application of artificial intelligence. Headquartered in the San Francisco Bay Area, Xaira combines expertise in machine learning, extensive biological and clinical data generation, and robust therapeutic product development. By harnessing advanced AI techniques, the company aims to enhance the understanding of biological processes and improve the effectiveness of therapies. Xaira's approach encompasses all stages of drug discovery, striving to transform how diseases are treated and enabling clients to deliver innovative therapeutic solutions.

Avenzo

Series A in 2024
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.

Regulus Therapeutics

Post in 2024
Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Comanche Biopharma

Series B in 2024
Comanche Biopharma is a biopharmaceutical company focused on developing an investigational small interfering RNA (siRNA) medicine aimed at treating preterm preeclampsia. The company is committed to ensuring that all women and their babies have access to safe and effective therapies for life-threatening complications during pregnancy. Comanche Biopharma's mission emphasizes the importance of evidence-based and affordable solutions, enabling women to receive cost-effective treatments that address the root causes of preeclampsia.

Nexo Therapeutics

Series A in 2023
Nexo Therapeutics is a biotechnology company focused on developing small-molecule therapies for cancer patients who have few effective treatment options. The company utilizes a specialized platform that integrates covalent ligand discovery with chemical biology to create innovative oncology drugs aimed at challenging targets that have been difficult to address in the past. By advancing its pipeline of novel therapies, Nexo Therapeutics seeks to provide new avenues for treatment, ultimately enhancing the care available to individuals battling cancer and other related diseases.

Gossamer Bio

Post in 2023
Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that focuses on the discovery and development of innovative therapeutic products for unmet medical needs. Founded by former executives from Receptos, the company aims to leverage a strong in-licensing strategy and a team with extensive experience in immunology, inflammation, fibrosis, and oncology. Gossamer Bio's pipeline includes several promising candidates: Seralutinib, which targets pulmonary arterial hypertension; GB004, aimed at treating inflammatory bowel diseases such as ulcerative colitis and Crohn's disease; GB1275, designed for various oncology indications; and GB001, intended for moderate-to-severe eosinophilic asthma. Through these efforts, Gossamer Bio seeks to improve patient outcomes in critical therapeutic areas.

Tagworks Pharmaceuticals

Series A in 2023
Tagworks Pharmaceuticals is a privately held biotech company based in the Netherlands. Tagworks develops In vivo chemistry to improve the efficacy of cancer therapies and to enable novel approaches in companion diagnostics. The company’s facilities are located at the Radboud University Medical Center in Nijmegen. Founded in 2011, Tagworks is a spin-out of Philips Healthcare, and originates from Philips’ molecular imaging expertise.

Regulus Therapeutics

Post in 2023
Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Fire1

Venture Round in 2023
FIRE1 and is based in Dublin, Ireland, will focus on developing novel therapeutic devices.

Launchpad Therapeutics

Series A in 2022
Launchpad Therapeutics operates as an antibody-focused precision oncology company.

Escient Pharmaceuticals

Series C in 2022
Escient Pharmaceuticals is a biotechnology company based in San Diego, California, that develops and manufactures drugs targeting G protein-coupled receptors (GPCRs) for the treatment of neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, the company specializes in harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs), to address various neurosensory-inflammatory disorders. Escient's innovative approach aims to provide first-in-class therapies for conditions associated with mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers and patients facing these challenging diseases.

MBX Biosciences

Series B in 2022
MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Inversago Pharma

Series C in 2022
Inversago Pharma Inc. is a preclinical-stage biotechnology company based in Montreal, Canada, focused on developing innovative therapies targeting metabolic and fibrotic disorders. The company specializes in peripherally-restricted CB1 receptor inverse agonists, which aim to offer new treatment options for conditions such as Prader-Willi syndrome, diabetes, and related metabolic disorders. Inversago Pharma's research includes addressing complications associated with diabetic kidney disease, non-alcoholic steatohepatitis, and various fibrotic diseases, including interstitial lung disease. Founded in 2015, the company is dedicated to enhancing patient outcomes through its pioneering approach to CB1 blockade.

Allakos

Post in 2022
Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company focuses on developing antibody-based therapeutics aimed at treating various conditions linked to dysregulation of the T-helper type 2 immune response, such as allergic diseases and chronic inflammation. Its lead product, antolimab (AK002), targets conditions including eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets Siglec-6, an inhibitory receptor found on mast cells. By binding to Siglec-6, AK006 is designed to enhance the receptor's natural inhibitory function, thereby reducing mast cell activation and associated inflammatory responses.

Moximed

Series C in 2022
Moximed, Inc. is a medical technology company based in Hayward, California, focused on developing innovative solutions for patients with knee osteoarthritis. The company offers the Atlas System, an implantable joint unloader designed to alleviate pain for individuals suffering from medial compartment knee osteoarthritis. This system operates similarly to a shock absorber, providing dynamic load reduction to minimize joint pain and enhance mobility. Moximed also features the MISHA Knee System, which offers an outpatient treatment option for patients who have not found relief through other therapies and are not suitable candidates for knee replacement surgery. By addressing the specific needs of osteoarthritis patients, Moximed aims to improve their quality of life while potentially delaying the need for more invasive surgical interventions. Founded in 2006, the company serves patients and medical professionals worldwide.

Gossamer Bio

Post in 2022
Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that focuses on the discovery and development of innovative therapeutic products for unmet medical needs. Founded by former executives from Receptos, the company aims to leverage a strong in-licensing strategy and a team with extensive experience in immunology, inflammation, fibrosis, and oncology. Gossamer Bio's pipeline includes several promising candidates: Seralutinib, which targets pulmonary arterial hypertension; GB004, aimed at treating inflammatory bowel diseases such as ulcerative colitis and Crohn's disease; GB1275, designed for various oncology indications; and GB001, intended for moderate-to-severe eosinophilic asthma. Through these efforts, Gossamer Bio seeks to improve patient outcomes in critical therapeutic areas.

Senti Bio

Post in 2022
Senti Biosciences is a clinical-stage biotechnology company specializing in synthetic biology and gene circuit engineering for therapeutic purposes. The company employs its Gene Circuits platform to develop innovative therapies that enhance precision in targeting cancer cells while avoiding damage to healthy cells. Senti Bio's approach involves the use of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells that incorporate its gene circuit technologies, aimed at addressing various oncology indications. Among its product candidates are SENTI-202, a Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells without harming healthy bone marrow, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences aims to provide effective treatment options for difficult-to-treat diseases through advanced cell and gene therapies.

Celcuity

Post in 2022
Celcuity Inc. is a clinical-stage biotechnology company based in Minneapolis, Minnesota, focused on advancing cancer treatment through innovative diagnostics. The company's CELsignia diagnostic platform utilizes living tumor cells to uncover specific cellular processes that drive cancer in individual patients. This approach enables the identification of new cancer sub-types that traditional molecular diagnostics may overlook, ultimately expanding therapeutic options. Celcuity is developing the CELsignia HSF test to diagnose two new sub-types of HER2-negative breast cancer and the CELsignia MP test for diagnosing 11 sub-types across various cancers, including breast, lung, colon, ovarian, kidney, and bladder. The company is also advancing its lead therapeutic candidate, gedatolisib, a pan-PI3K and mTOR inhibitor, currently being evaluated in a Phase 3 clinical trial for patients with advanced HR+/HER2- breast cancer.

Splice Bio

Series A in 2022
Splice Bio is a biotechnology company based in Barcelona, Spain, focused on developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012 and originally named Proteodesign, S.L., the company aims to address the needs of patients suffering from incurable genetic diseases. The intein platform, developed from research in the Muir Lab at Princeton University, seeks to overcome two significant limitations of traditional adeno-associated virus (AAV) therapies: it enhances the capacity for larger gene delivery and broadens the range of tissues that can be targeted for treatment.

Sorriso Pharmaceuticals

Series A in 2021
Sorriso Pharmaceuticals is a biotechnology company advancing a pipeline of disease-modifying antibodies for the treatment of inflammatory disease, including Crohn’s disease and ulcerative colitis. The Sorriso platform generates potent antibodies that can be delivered orally and are designed to maintain activity throughout the intestinal system. The company was founded in 2020 and is based in San Diego, California.

AviadoBio

Series A in 2021
AviadoBio is focused on developing innovative gene therapies aimed at transforming the lives of individuals affected by neurodegenerative disorders, specifically frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages advanced research from King's College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a comprehensive understanding of brain biology with proprietary gene therapy techniques, AviadoBio seeks to address the challenges of effectively delivering treatments to targeted areas in the brain. This approach aims to enhance the therapeutic potential of gene therapies, offering hope for halting or potentially reversing the progression of these debilitating diseases.

Tune Therapeutics

Series A in 2021
Tune Therapeutics is focused on developing innovative cell and gene therapies that utilize epigenetic programming to address serious diseases. By harnessing the epigenome, the company aims to create new pathways for treatment without making changes to the DNA sequence itself. This approach allows for the potential development of therapies for complex and widespread medical conditions, positioning Tune Therapeutics at the forefront of advancements in the field of epigenetics.

Regulus Therapeutics

Post in 2021
Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Recludix Pharma

Series A in 2021
Recludix Pharma is a biotechnology company focused on drug discovery, specifically targeting the development of potent and selective inhibitors for challenging protein targets associated with cancer and autoimmune diseases. The company utilizes a unique platform that combines custom-generated DNA-encoded libraries with massively parallel structure-activity relationship determination and proprietary screening tools. This integrated approach aims to enhance the identification and development of novel small molecule medicines, enabling healthcare professionals to create precision therapies that effectively address specific protein targets in these complex diseases.

Allay Therapeutics

Series C in 2021
Allay Therapeutics is focused on developing innovative pain management solutions aimed at improving post-surgical recovery for patients. The company utilizes a proprietary technology platform that integrates validated non-opioid analgesics with biopolymers to create dissolvable therapeutic products. These products are designed to deliver targeted pain relief at the surgical site over extended periods, significantly longer than conventional pain treatments. Allay's lead investigational product, ATX101, is currently undergoing evaluation in a Phase 2 clinical trial for total knee arthroplasty surgeries. With a team of experienced entrepreneurs, scientists, and clinicians based in the San Francisco Bay Area and Singapore, Allay Therapeutics is dedicated to transforming the approach to pain management and addressing the limitations associated with opioid use.

Shape Therapeutics

Series B in 2021
Shape Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Seattle, Washington. The company specializes in RNA-editing gene therapy targeting neurodegenerative disorders, oncology, metabolic conditions, and rare genetic diseases. Its proprietary RNAfix technology enables precise editing of RNA using the body's natural cellular mechanisms, which minimizes risks associated with immunogenicity, cellular toxicity, and off-target DNA editing. Shape Therapeutics employs a comprehensive approach to RNA gene therapy, integrating innovative payload, delivery, and manufacturing platform technologies. This strategy aims to facilitate the development of effective treatments and potential cures for a wide range of diseases, with the goal of enhancing patient outcomes across the healthcare landscape.

Stablix

Series A in 2021
Stablix Therapeutics is a biotechnology company specializing in Targeted Protein Stabilization (TPS). The company has developed a platform that produces heterobifunctional small molecules, known as RESTORACS, which selectively recruit deubiquitinase enzymes to remove ubiquitin from targeted proteins. This process enhances the stability and activity of these proteins. Stablix focuses on utilizing its innovative technology to create therapeutic programs aimed at treating rare diseases, cancer, and immunological disorders. By restoring protein function, Stablix aims to provide more effective treatment options for complex medical conditions.

Code Biotherapeutics

Seed Round in 2021
Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Monte Rosa Therapeutics

Series C in 2021
Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Satsuma Pharmaceuticals

Post in 2021
Satsuma Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.

Sagimet Biosciences

Venture Round in 2021
Sagimet Biosciences is a clinical-stage biopharmaceutical company based in San Mateo, California, that specializes in developing novel therapeutics for oncology and infectious diseases. The company focuses on targeting dysfunctional metabolic pathways, particularly through its expertise in fatty acid synthase (FASN) biology. Its lead drug candidate, denifanstat, is a selective FASN inhibitor designed for the treatment of nonalcoholic steatohepatitis (NASH) and is administered as an oral, once-daily pill. Additionally, Sagimet is working on antiviral therapeutics aimed at addressing respiratory syncytial virus, cytomegalovirus, and other human pathogens. Originally known as 3-V Biosciences, Inc., the company rebranded to Sagimet Biosciences in August 2019 and has been active since its incorporation in 2006.

Reneo Pharmaceuticals

Series B in 2020
Reneo Pharmaceuticals is a clinical-stage pharmaceutical company based in San Diego, California, that focuses on developing therapies for patients with rare genetic mitochondrial diseases. These conditions are linked to defects in energy metabolism, leading to issues such as muscle weakness and wasting. The company is developing REN001, an oral drug designed to modulate genes involved in mitochondrial function, aiming to enhance adenosine triphosphate (ATP) production, which is essential for cellular energy. By improving mitochondrial activity, Reneo seeks to enhance the daily functioning and quality of life for patients suffering from these disorders. Founded in 2014, Reneo Pharmaceuticals is committed to addressing the needs of individuals with orphan metabolic diseases.

Resilience

Series B in 2020
Resilience is a technology-driven biomanufacturing company focused on enhancing access to complex medicines, including biologics, vaccines, nucleic acids, and cell and gene therapies. The firm aims to transform the production of medicine by providing a comprehensive range of customized manufacturing capabilities that cater to the diverse needs of biopharmaceuticals throughout all stages of drug development, from pre-clinical to commercial supply. Resilience is committed to creating a sustainable network of advanced manufacturing solutions that ensure rapid, safe, and scalable production of treatments, thereby enabling partners to concentrate on breakthrough discoveries that improve patient outcomes. The company's innovative approach also strengthens biopharmaceutical supply chains, preparing them for future challenges.

Monte Rosa Therapeutics

Series B in 2020
Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

POLYNEURON

Series A in 2020
Polyneuron Pharmaceuticals AG is a biotechnology company based in Basel, Switzerland, founded in 2014. The company focuses on developing innovative therapeutics for antibody-mediated autoimmune disorders, particularly those affecting the peripheral nervous system. Its lead candidates include PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron employs a unique platform technology called Antibody-Catch, which utilizes biodegradable glycopolymers to selectively target and eliminate pathogenic autoantibodies while preserving the overall integrity of the immune system. This approach aims to provide safe and effective treatment options for patients suffering from these complex immune disorders.

Korro Bio

Series A in 2020
Korro Bio Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing nucleic acid-based therapeutics aimed at editing RNA to treat rare and prevalent diseases. Founded in 2018, the company has created a proprietary platform that allows for efficient and selective RNA editing, leveraging natural processes found in multicellular organisms. This innovative approach enables precise modifications to messenger RNA, allowing for targeted changes in protein structure and function across various tissues. By focusing on RNA instead of DNA, Korro Bio aims to enhance the precision and adaptability of genetic medicines, expanding their potential applications in treating a range of medical conditions.

MBX Biosciences

Series A in 2020
MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Annexon Biosciences

Series D in 2020
Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for patients suffering from classical complement-mediated disorders affecting the body, brain, and eye. The company's research is centered around its platform technology that targets classical complement-mediated autoimmune and neurodegenerative diseases associated with the abnormal activation of C1q, a key molecule in the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody intended for intravenous use in treating autoimmune and neurodegenerative disorders. Another candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration aimed at neurodegenerative ophthalmic conditions. The company employs a disciplined, biomarker-driven development strategy to ensure its therapies effectively engage the target at tolerable doses within the intended patient tissues, while also exploring additional indications within both orphan and larger market segments.

Monte Rosa Therapeutics

Series A in 2020
Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Amplyx Pharmaceuticals

Series C in 2020
Amplyx Pharmaceuticals is a pre-clinical stage company focusing on the development of small molecule drugs with enhanced efficacy and lower toxicity. Amplyx employs a platform approach to improve small molecule drugs by the addition of a second small molecule to an existing drug. The company develops innovative therapies for debilitating and life-threatening diseases that affect people with compromised immune systems. Amplyx Pharmaceuticals was founded in 2006 and is headquartered in San Diego, California.

Affinia Therapeutics

Series A in 2020
Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Akouos

Series B in 2020
Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Cardionomic

Venture Round in 2020
Cardionomic treats reduced cardiac output – ADHF’s root cause – by neuromodulating the terminal branches of the cardiac plexus to benignly increase contractility. Cardionomic’s acute, non-systemic therapy will improve cardiac output, renal blood flow, urine production, and pharmacological tolerance. Concurrently the therapy also achieves the associated decreases in plasma norepinephrine (NE) and B-type natriuretic peptide (BNP), which are objective biomarkers of chronic heart failure.

Inventiva Pharma

Post in 2020
Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies for patients with significant unmet medical needs, particularly in fibrosis, lysosomal storage disorders, and oncology. The company specializes in drugs that target nuclear receptors, transcription factors, and epigenetic modulation. Its primary product candidate, lanifibranor, is being developed for the treatment of non-alcoholic steatohepatitis (NASH), a condition currently lacking approved therapies. Inventiva has established a robust pipeline supported by a proprietary discovery engine, a dedicated research and development facility, and a skilled team with extensive experience in drug development. In addition to advancing lanifibranor, the company is also working on several earlier-stage programs aimed at addressing various medical conditions. All operations are conducted in France.

erytech

Post in 2020
ERYtech Pharma specializes in the encapsulation of therapeutic molecules and enzymes in red blood cells. It has achieved a major breakthrough in the fields of cancer and enzyme therapy by significantly increasing the therapeutic index of the encapsulated molecule or enzyme.

Geron

Post in 2020
Geron Corporation is a late-stage clinical biopharmaceutical company headquartered in Menlo Park, California, that specializes in developing therapies for hematologic myeloid malignancies. The company's lead product, imetelstat, is a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. This investigational drug aims to inhibit the uncontrolled proliferation of malignant progenitor cells, which can lead to dysfunctional blood cell production, thereby facilitating the recovery of normal blood cell development. Founded in 1990, Geron focuses on advancing treatments for conditions such as myelodysplastic syndromes and myelofibrosis, a rare blood cancer affecting bone marrow. The company also generates revenue through collaboration agreements, milestones, royalties, and licensing arrangements.

Black Diamond Therapeutics

Series C in 2019
Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

Shape Therapeutics

Series A in 2019
Shape Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Seattle, Washington. The company specializes in RNA-editing gene therapy targeting neurodegenerative disorders, oncology, metabolic conditions, and rare genetic diseases. Its proprietary RNAfix technology enables precise editing of RNA using the body's natural cellular mechanisms, which minimizes risks associated with immunogenicity, cellular toxicity, and off-target DNA editing. Shape Therapeutics employs a comprehensive approach to RNA gene therapy, integrating innovative payload, delivery, and manufacturing platform technologies. This strategy aims to facilitate the development of effective treatments and potential cures for a wide range of diseases, with the goal of enhancing patient outcomes across the healthcare landscape.

Sagimet Biosciences

Series E in 2019
Sagimet Biosciences is a clinical-stage biopharmaceutical company based in San Mateo, California, that specializes in developing novel therapeutics for oncology and infectious diseases. The company focuses on targeting dysfunctional metabolic pathways, particularly through its expertise in fatty acid synthase (FASN) biology. Its lead drug candidate, denifanstat, is a selective FASN inhibitor designed for the treatment of nonalcoholic steatohepatitis (NASH) and is administered as an oral, once-daily pill. Additionally, Sagimet is working on antiviral therapeutics aimed at addressing respiratory syncytial virus, cytomegalovirus, and other human pathogens. Originally known as 3-V Biosciences, Inc., the company rebranded to Sagimet Biosciences in August 2019 and has been active since its incorporation in 2006.

Vesper Medical

Venture Round in 2019
Vesper Medical is committed to developing safe, efficacious and easy-to-use products for patients with deep venous disease and the physicians who treat them. The company's core technology, the Vesper DUO Venous Stent System, is purpose-built for venous stenting, with an unmatched combination of strength and flexibility designed to meet the multiple anatomical challenges presented by vein mechanics. Vesper Medical is advancing the treatment of deep venous disease by creating a highly differentiated, purpose-built venous stent portfolio that addresses the multiple anatomical challenges presented by the iliac and femoral veins. It also develops minimally- invasive peripheral vascular products. Vesper Medical was founded in 2016 and is headquartered in Wayne, PA, USA.

Reneo Pharmaceuticals

Series A in 2019
Reneo Pharmaceuticals is a clinical-stage pharmaceutical company based in San Diego, California, that focuses on developing therapies for patients with rare genetic mitochondrial diseases. These conditions are linked to defects in energy metabolism, leading to issues such as muscle weakness and wasting. The company is developing REN001, an oral drug designed to modulate genes involved in mitochondrial function, aiming to enhance adenosine triphosphate (ATP) production, which is essential for cellular energy. By improving mitochondrial activity, Reneo seeks to enhance the daily functioning and quality of life for patients suffering from these disorders. Founded in 2014, Reneo Pharmaceuticals is committed to addressing the needs of individuals with orphan metabolic diseases.

Inozyme

Series A in 2019
Inozyme is a provider of therapies for rare diseases of calcification affecting soft tissues and bone created to offer potentially disease-modifying therapies to help children who are affected with rare, but severe and debilitating disorders of metabolism. The company's enzyme replacement therapy provides the treatment of Generalized Arterial Calcification of Infancy (GACI) and Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2), enabling patients to treat diseases characterized by mineral imbalances in the body.

Imara

Series B in 2019
Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing and commercializing therapeutics for rare genetic disorders of hemoglobin, including sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing its lead product candidate, IMR-687, which is a highly selective small molecule inhibitor of PDE9. IMR-687 is designed as an oral, once-a-day treatment that aims to modify disease through a multimodal mechanism of action affecting various cell types, including red and white blood cells. Imara is dedicated to addressing the unmet medical needs of patients suffering from these serious hemoglobinopathies.

Oyster Point

Series B in 2019
Oyster Point Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for ocular surface diseases. The company's primary focus is on its lead product candidate, OC-01, a nasal spray that acts as a selective nicotinic acetylcholine receptor agonist. OC-01 aims to address the signs and symptoms of dry eye disease, a chronic condition that affects over 30 million Americans. By activating the trigeminal parasympathetic pathway, OC-01 seeks to restore tear film homeostasis and stimulate the natural production of tears through the Lacrimal Functional Unit.

Black Diamond Therapeutics

Series B in 2019
Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

Lungpacer Medical

Venture Round in 2019
Lungpacer Medical Inc. is a medical device company specializing in therapeutic solutions aimed at enhancing the treatment of critically ill patients who require mechanical ventilation. Founded in 2009 and headquartered in Vancouver, Canada, with an additional office in Exton, Pennsylvania, the company has developed the Lungpacer DPT System. This diaphragm pacing system utilizes a temporary, minimally invasive, transvascular nerve stimulation technique to activate the diaphragm muscle, preserving its strength and integrity. By addressing the significant health and cost challenges associated with mechanical ventilation in Intensive Care Units, Lungpacer Medical aims to improve patient outcomes and reduce hospital care costs. The technology is designed to restore negative pressure ventilation, offering a more physiological respiratory pattern and mitigating the harmful effects associated with positive pressure ventilation.

Curzion Pharmaceuticals

Venture Round in 2019
Curzion Pharmaceuticals is a privately held development-stage biopharma company, and its development-stage oral selective lysophosphatidic acid 1 receptor (LPAR1) antagonist, CZN001 (renamed HZN-825).

Trevi Therapeutics

Series C in 2019
Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an oral extended-release formulation. The company is focused on treating serious neurologically mediated conditions, specifically chronic pruritus, chronic cough in patients with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in individuals with Parkinson's disease. Trevi is currently conducting a Phase 2b/3 clinical trial, known as the PRISM trial, for nalbuphine ER, targeting severe pruritus associated with prurigo nodularis. The conditions being addressed share similar underlying mechanisms that involve opioid receptors in both the central and peripheral nervous systems. Founded in 2011, Trevi Therapeutics aims to provide innovative treatment options for patients suffering from these challenging health issues.

Allay Therapeutics

Series B in 2019
Allay Therapeutics is focused on developing innovative pain management solutions aimed at improving post-surgical recovery for patients. The company utilizes a proprietary technology platform that integrates validated non-opioid analgesics with biopolymers to create dissolvable therapeutic products. These products are designed to deliver targeted pain relief at the surgical site over extended periods, significantly longer than conventional pain treatments. Allay's lead investigational product, ATX101, is currently undergoing evaluation in a Phase 2 clinical trial for total knee arthroplasty surgeries. With a team of experienced entrepreneurs, scientists, and clinicians based in the San Francisco Bay Area and Singapore, Allay Therapeutics is dedicated to transforming the approach to pain management and addressing the limitations associated with opioid use.

Xenon Pharmaceuticals

Post in 2019
Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Burnaby, Canada, focused on developing therapeutics for neurological disorders. The company's clinical pipeline includes several product candidates aimed at treating epilepsy, such as XEN496 and XEN1101, both Kv7 potassium channel modulators currently in Phase II trials, and XEN901, a selective Nav1.6 sodium channel inhibitor in Phase I trials. Additionally, Xenon is developing XEN007, a central nervous system-acting calcium channel modulator, also in Phase I trials. The company leverages insights from genetic research, particularly from families with severe phenotypes, to identify drug targets, which may extend beyond rare genetic disorders. Furthermore, Xenon has established a collaboration with Neurocrine Biosciences to advance the development of innovative epilepsy treatments.

Annexon Biosciences

Series C in 2018
Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for patients suffering from classical complement-mediated disorders affecting the body, brain, and eye. The company's research is centered around its platform technology that targets classical complement-mediated autoimmune and neurodegenerative diseases associated with the abnormal activation of C1q, a key molecule in the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody intended for intravenous use in treating autoimmune and neurodegenerative disorders. Another candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration aimed at neurodegenerative ophthalmic conditions. The company employs a disciplined, biomarker-driven development strategy to ensure its therapies effectively engage the target at tolerable doses within the intended patient tissues, while also exploring additional indications within both orphan and larger market segments.

Qpex Biopharma

Series A in 2018
Qpex Biopharma develops a pipeline of agents addressing critical needs for the treatment of infectious diseases. It develops multiple antibiotic drugs intended to combat antimicrobial resistance. The company's technology is focused on targeting the treatment of infectious diseases for both inpatient and outpatient cases, deep expertise in the discovery, development and regulatory approval of anti-infective medicines, including an extensive record of working with public/private partnerships focused on new antibiotics to combat antimicrobial resistance, enabling medical businesses optimal use of products and critical needs for the treatment of infectious diseases in the inpatient and outpatient settings. It was founded in 2018 and is headquartered in San Diego, California.

PhaseBio

Series D in 2018
PhaseBio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for endocrine and metabolic disorders, as well as cardiovascular diseases. The company leverages its proprietary elastin-like polypeptides (ELPs) technology to enhance the stability, bioavailability, and efficacy of proteins and peptides. Its lead candidate, PB1023, is in a phase 2b clinical trial for managing hyperglycemia in type 2 diabetes, with potential applications in obesity. Additionally, PhaseBio is advancing Vasomera, a candidate in phase 1 trials aimed at treating pulmonary arterial hypertension and heart failure. The company is also exploring PB2452, a novel reversal agent for the antiplatelet drug ticagrelor, and PB1046 for pulmonary arterial hypertension. With a commitment to addressing unmet medical needs, PhaseBio is headquartered in Malvern, Pennsylvania.

Millendo Therapeutics

Venture Round in 2018
Millendo Therapeutics is a biopharmaceutical company dedicated to developing innovative treatments for endocrine diseases resulting from hormone dysregulation. The company focuses on addressing significant unmet medical needs by creating distinct therapies that improve clinical care. By leveraging scientific advancements, Millendo aims to develop novel compounds that can effectively treat complex conditions, ultimately enhancing the quality of life for patients, families, and caregivers.

Metacrine

Series C in 2018
Metacrine is a clinical-stage biopharmaceutical company focused on building an innovative pipeline of best-in-class drugs to treat liver and gastrointestinal (GI) diseases. The most advanced program is focused on the farnesoid X receptor (FXR) an important drug target in multiple liver and GI diseases. Beyond the FXR program, a pipeline of novel drug candidates against other drug targets is being explored by taking advantage of internal drug discovery and development capabilities.

MEI Pharma

Post in 2018
MEI Pharma, Inc. is a late-stage pharmaceutical company based in San Diego, California, concentrating on the clinical development of innovative cancer therapies. The company's pipeline includes Zandelisib, an oral phosphatidylinositol 3-kinase delta inhibitor currently undergoing Phase II trials for relapsed or refractory follicular lymphoma and Phase Ib trials for B-cell malignancies. Another key candidate is Voruciclib, an oral cyclin-dependent kinase inhibitor in Phase Ib trials for acute myeloid leukemia and B-cell malignancies. Additionally, MEI Pharma is developing ME-344, a mitochondrial inhibitor targeting HER2-negative breast cancer, currently in Phase I trials. Pracinostat, an oral histone deacetylase inhibitor, is also in Phase II trials aimed at treating myelodysplastic syndrome. The company has established strategic collaborations and licensing agreements with various organizations to enhance its research and development efforts. Founded in 2000, MEI Pharma was previously known as Marshall Edwards, Inc. and rebranded in 2012.

Senti Bio

Series A in 2018
Senti Biosciences is a clinical-stage biotechnology company specializing in synthetic biology and gene circuit engineering for therapeutic purposes. The company employs its Gene Circuits platform to develop innovative therapies that enhance precision in targeting cancer cells while avoiding damage to healthy cells. Senti Bio's approach involves the use of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells that incorporate its gene circuit technologies, aimed at addressing various oncology indications. Among its product candidates are SENTI-202, a Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells without harming healthy bone marrow, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences aims to provide effective treatment options for difficult-to-treat diseases through advanced cell and gene therapies.

CTI BioPharma

Post in 2018
CTI BioPharma Corp. is a biopharmaceutical company headquartered in Seattle, Washington, that specializes in the development and commercialization of targeted therapies for blood-related cancers. The company is advancing its lead product, pacritinib, an investigational oral kinase inhibitor currently in Phase III clinical trials for myelofibrosis, a type of bone marrow cancer. In addition to pacritinib, CTI BioPharma's development portfolio includes Pixantrone, which is in a Phase III trial for non-Hodgkin's lymphoma, and Brostallicin, currently undergoing a Phase II study for sarcoma. The company has established collaboration and licensing agreements with various pharmaceutical firms, including Teva Pharmaceutical Industries Ltd. and Novartis International Pharmaceutical, Ltd. Founded in 1991, CTI BioPharma aims to deliver innovative and less toxic cancer therapies to patients.

Fire1

Series C in 2018
FIRE1 and is based in Dublin, Ireland, will focus on developing novel therapeutic devices.

Centrexion Therapeutics

Series D in 2018
Centrexion Therapeutics Corporation is a late clinical-stage biopharmaceutical company based in Boston, Massachusetts, dedicated to developing non-opioid and non-addictive therapies aimed at addressing chronic pain. The company's product pipeline includes CNTX-4975, currently in Phase III trials for moderate to severe knee osteoarthritis pain, and in Phase II trials for Morton’s neuroma and canine osteoarthritis. Additionally, CNTX-0290 is in Phase I trials targeting chronic pain associated with inflammatory, neuropathic, and mixed conditions. Other candidates under development include CNTX-6970, for inflammatory pain, and CNTX-2022, a high-concentration topical gel formulation of lidocaine for various types of pain. CNTX-6016 is also in pre-clinical stages focusing on chronic neuropathic pain. Founded in 2013, Centrexion Therapeutics aims to provide safe and effective solutions for patients suffering from chronic pain conditions.

Korro Bio

Seed Round in 2018
Korro Bio Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing nucleic acid-based therapeutics aimed at editing RNA to treat rare and prevalent diseases. Founded in 2018, the company has created a proprietary platform that allows for efficient and selective RNA editing, leveraging natural processes found in multicellular organisms. This innovative approach enables precise modifications to messenger RNA, allowing for targeted changes in protein structure and function across various tissues. By focusing on RNA instead of DNA, Korro Bio aims to enhance the precision and adaptability of genetic medicines, expanding their potential applications in treating a range of medical conditions.

Pionyr Immunotherapeutics

Series B in 2017
Pionyr Immunotherapeutics Inc. is a biotechnology company based in San Francisco, California, that focuses on developing innovative cancer immunotherapies. Founded in 2015, the company aims to enhance the body's antitumor immunity by targeting the tumor microenvironment. Utilizing advanced target discovery and antibody generation technologies, Pionyr has created a unique approach called Myeloid Tuning™, which modifies the cellular infiltrate within tumors to improve the immune system's response against cancer. This strategy is intended to be particularly effective when used in conjunction with checkpoint inhibitors, offering new avenues for cancer treatment.

Metacrine

Series B in 2017
Metacrine is a clinical-stage biopharmaceutical company focused on building an innovative pipeline of best-in-class drugs to treat liver and gastrointestinal (GI) diseases. The most advanced program is focused on the farnesoid X receptor (FXR) an important drug target in multiple liver and GI diseases. Beyond the FXR program, a pipeline of novel drug candidates against other drug targets is being explored by taking advantage of internal drug discovery and development capabilities.

Akouos

Seed Round in 2017
Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Inozyme

Series A in 2017
Inozyme is a provider of therapies for rare diseases of calcification affecting soft tissues and bone created to offer potentially disease-modifying therapies to help children who are affected with rare, but severe and debilitating disorders of metabolism. The company's enzyme replacement therapy provides the treatment of Generalized Arterial Calcification of Infancy (GACI) and Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2), enabling patients to treat diseases characterized by mineral imbalances in the body.

Oyster Point

Series A in 2017
Oyster Point Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for ocular surface diseases. The company's primary focus is on its lead product candidate, OC-01, a nasal spray that acts as a selective nicotinic acetylcholine receptor agonist. OC-01 aims to address the signs and symptoms of dry eye disease, a chronic condition that affects over 30 million Americans. By activating the trigeminal parasympathetic pathway, OC-01 seeks to restore tear film homeostasis and stimulate the natural production of tears through the Lacrimal Functional Unit.

Cydan

Venture Round in 2017
Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The organization identifies and mitigates the risks associated with compounds that have therapeutic and commercial potential. Through a rigorous process, Cydan generates data to support the development of new therapies and the formation of new companies. Their experienced team leverages strong relationships with academia and patient advocacy groups, along with deep expertise in drug development and a proven track record of successful product commercialization. By guiding therapies through clinical, regulatory, and commercial development, Cydan aims to improve the lives of patients and families affected by rare diseases.

Amplyx Pharmaceuticals

Series C in 2017
Amplyx Pharmaceuticals is a pre-clinical stage company focusing on the development of small molecule drugs with enhanced efficacy and lower toxicity. Amplyx employs a platform approach to improve small molecule drugs by the addition of a second small molecule to an existing drug. The company develops innovative therapies for debilitating and life-threatening diseases that affect people with compromised immune systems. Amplyx Pharmaceuticals was founded in 2006 and is headquartered in San Diego, California.

Complexa

Series C in 2017
Complexa, Inc. is a biopharmaceutical company based in Berwyn, Pennsylvania, focused on developing treatments for severe and life-threatening diseases related to fibrosis and inflammation. Established in 2008, the company is advancing its lead compound, CXA-10, an oral nitrated fatty acid aimed at treating focal segmental glomerulosclerosis (FSGS) and pulmonary arterial hypertension (PAH). Complexa utilizes proprietary technologies that involve the synthesis of endogenous nitro-fatty acids, which serve as cell-signaling agents regulating key inflammatory pathways. This approach aims to enhance the body's natural mechanisms for resolving and repairing acute and chronic tissue injuries, with a focus on addressing various inflammatory, fibrotic, and central nervous system diseases.

Trevi Therapeutics

Series C in 2017
Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an oral extended-release formulation. The company is focused on treating serious neurologically mediated conditions, specifically chronic pruritus, chronic cough in patients with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in individuals with Parkinson's disease. Trevi is currently conducting a Phase 2b/3 clinical trial, known as the PRISM trial, for nalbuphine ER, targeting severe pruritus associated with prurigo nodularis. The conditions being addressed share similar underlying mechanisms that involve opioid receptors in both the central and peripheral nervous systems. Founded in 2011, Trevi Therapeutics aims to provide innovative treatment options for patients suffering from these challenging health issues.

Nightstar Therapeutics

Series C in 2017
NightstaRx Ltd. is a U.K. startup company. Gene therapy is the use of a gene to treat disease. The approach could enable treatments for a several rare conditions caused by mutations in a single gene.

OrphoMed

Series A in 2017
OrphoMed, Inc. is a San Francisco-based pharmaceutical company that specializes in developing innovative therapies for conditions such as irritable bowel syndrome with diarrhea (IBS-D), acute and chronic pain, and cystic fibrosis. The company utilizes its proprietary DIMERx™ platform technology to create novel therapeutic dimer conjugates that address visceral hypersensitivity and pancreatitis, particularly in gastrointestinal disorders and pain management. Its lead candidate, ORP-101, is designed as a peripherally acting sodium ion channel blocker that offers potential benefits without the abuse potential associated with traditional opioids. ORP-101 is positioned for two distinct indications: an oral form targeting colonic hyperalgesia and gastric dysmotility in IBS-D, and a parenteral form for treating peripheral neuropathic pain. OrphoMed is also developing additional non-opioid and opioid conjugates aimed at treating acute and chronic pain, with a focus on safety and reduced dependence risk. The components of these conjugates are based on previously validated compounds, which helps to lower developmental risks while providing new treatment options for patients.

Synlogic

Series C in 2017
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

EPIX Therapeutics

Venture Round in 2017
EPIX Therapeutics, Inc. is a medical device company based in Santa Clara, California, specializing in the development of advanced temperature-sensing irrigated radiofrequency ablation catheters for the treatment of atrial fibrillation and other cardiac arrhythmias. Utilizing its proprietary TempaSure technology, which incorporates microwave radiometry, EPIX Therapeutics provides precise real-time feedback on volumetric temperature during ablation procedures. This technology enables physicians to control and predict lesion formation more accurately, ultimately enhancing clinical outcomes for patients. Founded in 2007 and previously known as Advanced Cardiac Therapeutics, EPIX Therapeutics has formed strategic partnerships with industry leaders such as Abbott Laboratories and Hansen Medical Inc. The company operates as a subsidiary of Medtronic plc, focusing on improving the treatment of irregular heart rhythms through its innovative catheter-based systems.

Addex Therapeutics

Post in 2017
Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, that specializes in the discovery, development, and commercialization of small-molecule drugs targeting central nervous system (CNS) disorders. The company primarily focuses on allosteric modulators, a novel class of orally available drugs that interact with G-protein coupled receptors, potentially offering advantages over traditional therapeutic approaches. Its lead product candidates include Dipraglurant for treating Parkinson's disease-related dyskinesia and dystonia, ADX71149 for epilepsy and other CNS disorders, and GABAB PAM for addiction. Addex has established a collaboration with Janssen Pharmaceuticals Inc. to develop mGluR2PAM compounds aimed at addressing various health issues. Founded in 2002, Addex Therapeutics continues to advance its innovative drug discovery platform to improve therapeutic outcomes for patients with neurological conditions.

Rhythm Pharmaceuticals

Venture Round in 2017
Rhythm is a biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic disorders. Their lead peptide product candidate, setmelanotide, is a potent, first-in-class melanocortin-4 receptor (MC4R) agonist for the treatment of rare genetic disorders of obesity. They believe that setmelanotide, for which they have exclusive worldwide rights, has the potential to serve as replacement therapy for the treatment of melanocortin 4, or MC4, pathway deficiencies. The MC4 pathway is a compelling target for treating these genetic disorders because of its critical role in regulating appetite and weight, and peptide therapeutics are uniquely suited to activating this target.

Tarveda Therapeutics

Series D in 2017
Tarveda Therapeutics is a developer of miniature drug conjugates designed for the treatment of patients with a wide range of solid tumors. The company's HSP90 targeted drug conjugate platform with lead drug candidate PEN-866 selectively binds in tumors to the activated form of Heat Shock Protein 90 (HSP90) and releases its potent topoisomerase 1 inhibitor payload, SN38 and it also develops a miniature drug conjugate in clinical evaluation for the treatment of patients with somatostatin receptor 2 (SSTR2) expressing neuroendocrine, small cell lung and other solid tumors, enabling companies to extend the lives of patients with hard to treat cancers while minimizing potential toxicities.

Regulus Therapeutics

Post in 2017
Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Immunomedics

Post in 2017
Immunomedics is a biopharmaceutical company that specializes in developing monoclonal antibody-based products for the treatment of cancer and other serious diseases. Its corporate objective is to become a fully-integrated biopharmaceutical company. The company was founded in 1982 and headquartered in Morris Plains, New Jersey.

Trillium Therapeutics

Post in 2017
Trillium Therapeutics Inc. is a clinical-stage immuno-oncology company focused on developing innovative therapies for cancer treatment. The company's lead candidate, TTI-621, is a SIRPaFc fusion protein currently undergoing Phase I clinical trials for advanced relapsed or refractory hematologic malignancies, as well as solid tumors and mycosis fungoides. Another product, TTI-622, is also in Phase I clinical trials and is based on an immunoglobulin G4 SIRPaFc protein. Additionally, Trillium is exploring TTI-10001, a small molecule stimulator of interferon genes agonist in the discovery phase. Founded in 2004 and headquartered in Mississauga, Canada, Trillium Therapeutics has evolved from its previous name, Stem Cell Therapeutics Corp., to focus primarily on harnessing the immune system to combat cancer. The company operates across the United States and Canada, emphasizing the development of targeted protein therapeutics.

Cleave Therapeutics

Series B in 2016
Cleave Therapeutics develops oncology pharmaceuticals designed to target neurodengeneration and pathways used by cancer cells to grow. The company is engaged in the discovery of novel, small-molecule drugs that affect protein-degradation pathways, upon which cancer cells depend for survival, enabling patients to get cured from cancer.

VytronUS

Series C in 2016
VytronUS, Inc. specializes in the manufacture and marketing of cardiac medical devices aimed at treating cardiac arrhythmias. Founded in 2006 and located in Sunnyvale, California, the company focuses on leveraging ultrasound energy to enhance the imaging and therapeutic capabilities of its devices. These innovative products allow for precise non-contact energy delivery and facilitate the production of high-resolution maps of cardiac anatomy. By providing physicians with tools that enable visualization, precision, flexibility, and automation, VytronUS aims to improve the treatment of complex arrhythmias.

Vtesse

Series A in 2016
Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is conducting clinical studies for VTS-270, aimed at treating Niemann-Pick disease type C (NPC), and is also engaged in pre-clinical discovery and development of additional therapies for NPC and other lysosomal storage diseases. Vtesse originated as the first spin-out from Cydan Development, an orphan-drug accelerator that identifies promising therapeutic programs. The company collaborates with the National Institutes of Health (NIH) to advance its clinical studies and is supported by a consortium of experienced investors. Vtesse's management team has a strong background, having participated in the development of over 20 approved drugs and vaccines, which underscores its commitment to addressing the needs of underserved patient populations.

Ardelyx

Post in 2016
Ardelyx, Inc. is a biopharmaceutical company specializing in the development and commercialization of innovative oral therapeutics for cardiorenal diseases. Founded in 2007 and headquartered in Fremont, California, the company focuses on addressing significant unmet medical needs through first-in-class medicines. Its lead product candidate, tenapanor, has completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and hyperphosphatemia in end-stage renal disease patients on dialysis. Additionally, Ardelyx is advancing RDX013, a small molecule potassium secretagogue targeting hyperkalemia, and has other candidates in its pipeline, including RDX002 and RDX009, which are in preclinical development for chronic kidney disease and type 2 diabetes, respectively. Ardelyx employs a proprietary drug discovery platform aimed at creating non-systemic products that target specific transporters and receptors to minimize side effects associated with systemic exposure.

Annexon Biosciences

Series B in 2016
Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for patients suffering from classical complement-mediated disorders affecting the body, brain, and eye. The company's research is centered around its platform technology that targets classical complement-mediated autoimmune and neurodegenerative diseases associated with the abnormal activation of C1q, a key molecule in the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody intended for intravenous use in treating autoimmune and neurodegenerative disorders. Another candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration aimed at neurodegenerative ophthalmic conditions. The company employs a disciplined, biomarker-driven development strategy to ensure its therapies effectively engage the target at tolerable doses within the intended patient tissues, while also exploring additional indications within both orphan and larger market segments.

Verona Pharma

Post in 2016
Verona Pharma is a clinical-stage biopharmaceutical company based in London, established in 2005. The company specializes in developing and commercializing therapies for respiratory diseases that have significant unmet medical needs, including chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. Its lead product candidate, ensifentrine, is an inhaled dual inhibitor of the enzymes phosphodiesterase 3 and 4, functioning as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is being developed in various formulations, including nebulized, dry powder inhaler, and pressurized metered-dose inhaler, with the nebulized form currently in Phase 2b clinical development for the maintenance treatment of COPD. Verona Pharma's commitment is to enhance the health and quality of life for individuals suffering from chronic respiratory conditions through innovative therapeutic solutions.
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