Investor directory

Wellington Management

Wellington Management Company LLP, founded in 1928 and headquartered in Boston, Massachusetts, is a prominent investment management firm providing comprehensive solutions to institutional clients worldwide. The firm specializes in a diverse range of investment strategies, including equity, fixed income, multi-asset, and alternative investments, tailored to meet the unique return and risk objectives of its clients. Wellington Management employs a collaborative approach, leveraging proprietary research and analytical tools to identify opportunities and manage risks across global capital markets. The firm caters to a wide array of clients, including sovereign institutions, endowments, foundations, wealth managers, and insurance companies, spanning more than 50 countries. With a focus on long-term investment principles, Wellington Management fosters independent thought and debate within its teams, ensuring that resources are effectively utilized to deliver consistent results. The firm also emphasizes environmental, social, and governance (ESG) considerations in its research and investment processes, engaging with companies on key ESG topics.

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280 Congress Street, Boston, Massachusetts 02210, US

Pamela Choney

Partner and SVP

Anita M. Killian

Director, Partner, VP, Global Industry Analyst and Portfolio Manager

Past deals in Therapeutics

Boundless Bio

Series C in 2023
Boundless Bio, Inc., biopharma company, develops therapies and medicines for the treatment of cancers. It offers therapeutics based on extrachromosomal DNA (ecDNA) one of the drivers of the aggressive cancers, such as cancers characterized by high copy number amplification of oncogenes. The company was formerly known as Pretzel Therapeutics, Inc. Boundless Bio, Inc. was founded in 2018 and is based in LA Jolla, California.

Acrivon Therapeutics

Series B in 2021
Acrivon Therapeutics is a biotech company specializing in drug development through early clinical treatment success.

Ventyx Biosciences

Series B in 2021
Ventyx Biosciences, Inc. is a biotechnology company based in Encinitas, California, focused on developing selective inhibitors of TYK2 for the treatment of autoimmune diseases. Incorporated in 2018, the company boasts a diverse portfolio of innovative clinical and pre-clinical programs aimed at high-value therapeutic targets. Through its research and development efforts, Ventyx is committed to advancing treatments that address unmet medical needs in the autoimmune disease space.

NiKang Therapeutics

Series C in 2021
NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.

Nuvalent

Series B in 2021
Nuvalent is a biotechnology company that develops targeted therapies for clinically proven kinase targets in cancer. Leveraging deep expertise in structure-based design, Nuvalent develops innovative small molecules with exquisite target selectivity to overcome resistance, minimize adverse events, and drive more durable responses. It is advancing a robust pipeline with parallel lead programs in ROS1-positive and ALK-positive NSCLC, along with multiple discovery-stage research programs. It was founded in 2017 and is based in Cambridge, Massachusetts.

Boundless Bio

Series B in 2021
Boundless Bio, Inc., biopharma company, develops therapies and medicines for the treatment of cancers. It offers therapeutics based on extrachromosomal DNA (ecDNA) one of the drivers of the aggressive cancers, such as cancers characterized by high copy number amplification of oncogenes. The company was formerly known as Pretzel Therapeutics, Inc. Boundless Bio, Inc. was founded in 2018 and is based in LA Jolla, California.

Entrada Therapeutics

Series B in 2021
Entrada Therapeutics, Inc., a biotechnology company, engages in the treatment of diseases through the intracellular delivery of biologic. The company enables intracellular delivery of proteins, peptides, and nucleic acids and allows development of programs across intracellular target classes. Its Intracellular enzyme replacement therapy (IC-ERT) is a medical treatment that corrects an enzyme deficiency in the cell, Protein-Protein Interaction Inhibitors. The company was founded in 2016 and is based in Boston, Massachusetts.

Scribe Therapeutics

Series B in 2021
Scribe Therapeutics Inc. engages in the engineering, delivery, and development of in vivo therapies and CRISPR molecules to rewrite and repair the underlying cause of genetic disorders. Its technology includes X-Editing (XE), an engineered molecule for therapeutic use and in vivo genetic modification. The company was incorporated in 2017 and is based in Berkeley, California.

Artiva Biotherapeutics

Series B in 2021
Artiva Biotherapeutics, Inc., a biotech company, develops and manufactures cellular immunotherapies for cancer patients. The company offers a pipeline of off-the-shelf, allogeneic natural killer (NK) cell therapies for the treatment of hematologic cancers or solid tumors. The company’s products target CD20 and CD19 in B-cell lymphomas and HER2 in various solid tumors. The company was founded in 2019 and is headquartered in San Diego, California. Artiva Biotherapeutics, Inc. operates as a subsidiary of Green Cross Holdings Corporation.

Centessa Pharmaceuticals

Series A in 2021
Centessa Pharmaceuticals is a next-generation biopharmaceutical company that aims to reshape the traditional drug development process. The company applies an asset-centric R&D model at scale to advance a portfolio of highly validated programs led by industry-leading teams. Each program is developed by a Centessa Subsidiary and supported by a centralized infrastructure and the Centessa management team.

Gemini Therapeutics

Post in 2021
Gemini Therapeutics, Inc. is a precision medicine company established in 2015 and based in Cambridge, Massachusetts. The company specializes in developing therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini's approach focuses on addressing genetically-defined conditions by creating therapeutic candidates that target specific molecular abnormalities prevalent in patients with significant clinical needs. Their diverse pipeline features a range of innovative treatment modalities, including monoclonal antibodies, recombinant proteins, and gene therapies. The company has secured funding from prominent life science investors and collaborates with academic institutions globally to advance its mission of delivering potentially first-in-class therapeutics.

Design Therapeutics

Series B in 2021
Design Therapeutics, Inc. develops therapies for the treatment of degenerative disorders caused by nucleotide repeat expansions. The company engages in the development of a program for the treatment of Friedreich’s ataxia and degenerative diseases such as Fragile X syndrome and myotonic dystrophy. The company was incorporated in 2017 and is based in Solana Beach, California.

Verve Therapeutics

Series B in 2021
Verve Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to reduce the risk of coronary artery disease, a leading cause of death globally. Founded in 2018 and headquartered in Cambridge, Massachusetts, with a research facility in Philadelphia, the company leverages human genetic analysis and gene-editing technologies to create treatments that offer lifelong protection from this condition. Verve has established strategic alliances, including collaborations with Beam Therapeutics to advance delivery technologies targeting cardiovascular issues and with Verily for developing gene editing delivery vehicles. Formerly known as Endcadia, Verve Therapeutics rebranded in September 2019 to reflect its commitment to addressing heart disease through advanced genetic solutions.

Scorpion Therapeutics

Series B in 2021
Scorpion Therapeutics, Inc. is a biotechnology company based in Boston, Massachusetts, focused on developing advanced precision oncology therapies for cancer treatment. Founded in 2020, the company aims to enhance the accessibility and effectiveness of precision medicine for cancer patients. Scorpion Therapeutics specializes in creating drugs in three primary areas: therapies targeting known oncogenes, agents for currently undruggable cancer targets, and innovative drugs for new targets. By integrating state-of-the-art technologies in target discovery, medicinal chemistry, and translational medicine, the company is committed to delivering next-generation precision oncology solutions. Its experienced leadership team is dedicated to employing a tailored approach to efficiently advance the field of precision cancer treatment.

Edgewise Therapeutics

Series C in 2020
Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, specializing in the development of small molecule therapies for severe musculoskeletal diseases. Founded in 2017, the company leverages its expertise in skeletal muscle physiology to create innovative treatments for genetically defined muscle disorders, including Duchenne, Becker, and limb girdle muscular dystrophies. Edgewise Therapeutics employs a precision medicine platform to identify and target key muscle proteins and modulators, aiming to address the underlying causes of these conditions. Additionally, the company has established a state-of-the-art research facility to enhance its capabilities in understanding muscle adaptation and injury related to disease.

LianBio

Venture Round in 2020
LianBio’s mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and major Asian markets through partnerships that provide access to the best science-driven therapeutic discoveries. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a pipeline based on disease relevance and the ability to impact patients with transformative mechanisms and precision-based therapeutics.

Gracell Biotechnologies

Series C in 2020
Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company based in Shanghai, China, focused on discovering and developing innovative cell therapies for cancer treatment. The company's lead product candidates include GC012F, a dual-targeted CAR-T therapy for multiple myeloma currently in Phase I trials; GC019F, which targets adult B cell acute lymphoblastic leukemia and is also in Phase I trials; and GC007F, aimed at B cell non-Hodgkin’s lymphoma, also in Phase I. Additionally, Gracell is developing GC027, a CAR-T candidate targeting adult T cell acute lymphoblastic leukemia, and GC007g, an allogeneic CAR-T therapy for relapsed or refractory B-ALL, both in Phase I trials. Beyond these, Gracell has a pipeline of earlier-stage product candidates targeting various cancers, including ovarian and breast cancer. Founded in 2017, Gracell Biotechnologies is committed to advancing cellular therapeutics to improve patient outcomes in hematological malignancies and solid tumors.

Nuvation Bio

Post in 2020
Nuvation Bio Inc., a biopharmaceutical company, focuses on the development of therapies for oncology. Its portfolio includes various oncology programs with multiple drug development candidates. Nuvation Bio Inc.was formerly known as RePharmation Inc. and changed its name to Nuvation Bio Inc. in April 2019. The company was founded in 2018 and is based in New York, New York with an additional office in San Francisco, California.

Olema Oncology

Series C in 2020
Olema Oncology is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapies for women's cancers, particularly focusing on estrogen receptor-positive breast cancer. The company's lead candidate, OP-1250, is an estrogen receptor antagonist and selective degrader, currently undergoing Phase 1/2 clinical trials for the treatment of metastatic or locally advanced, estrogen receptor-positive, and human epidermal growth factor receptor 2-negative breast cancer. Olema Oncology aims to innovate targeted therapies by leveraging its expertise in endocrine-driven cancers and mechanisms of acquired resistance. The company seeks to enhance treatment options for both pre- and post-menopausal women with breast cancer, striving to transform the standard of care with more effective and convenient therapies. Founded in 2006 and headquartered in San Francisco, California, Olema was previously known as CombiThera, Inc. and rebranded in 2009.

Dyne Therapeutics

Series B in 2020
Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It develops various programs for myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapies. The company was founded in 2017 and is based in Waltham, Massachusetts.

PMV Pharmaceutcals

Series D in 2020
PMV Pharma is developing first-in-class p53 and p53 pathway modulators for the treatment of cancer. Bringing together leaders in the field to utilize over three decades of p53 biology, PMV Pharma combines unique biological understanding with pharmaceutical development focus.

Olema Oncology

Series B in 2020
Olema Oncology is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapies for women's cancers, particularly focusing on estrogen receptor-positive breast cancer. The company's lead candidate, OP-1250, is an estrogen receptor antagonist and selective degrader, currently undergoing Phase 1/2 clinical trials for the treatment of metastatic or locally advanced, estrogen receptor-positive, and human epidermal growth factor receptor 2-negative breast cancer. Olema Oncology aims to innovate targeted therapies by leveraging its expertise in endocrine-driven cancers and mechanisms of acquired resistance. The company seeks to enhance treatment options for both pre- and post-menopausal women with breast cancer, striving to transform the standard of care with more effective and convenient therapies. Founded in 2006 and headquartered in San Francisco, California, Olema was previously known as CombiThera, Inc. and rebranded in 2009.

Freeline

Series C in 2020
Freeline Therapeutics is a clinical-stage biotechnology company focused on developing liver-directed gene therapies for bleeding disorders and other diseases. Based in Stevenage, United Kingdom, Freeline's lead product candidate, FLT180a, is undergoing Phase 1/2 clinical trials aimed at treating moderate to severe hemophilia B. The company's pipeline also includes FLT190, which is in dose-escalating Phase 1/2 trials for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A. Freeline utilizes a next-generation proprietary adeno-associated virus (AAV) vector platform, enhancing its potential for systemic gene therapy applications. Since its founding in 2015, the company has been dedicated to advancing innovative treatments in the field of gene therapy.

Verve Therapeutics

Series A in 2020
Verve Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to reduce the risk of coronary artery disease, a leading cause of death globally. Founded in 2018 and headquartered in Cambridge, Massachusetts, with a research facility in Philadelphia, the company leverages human genetic analysis and gene-editing technologies to create treatments that offer lifelong protection from this condition. Verve has established strategic alliances, including collaborations with Beam Therapeutics to advance delivery technologies targeting cardiovascular issues and with Verily for developing gene editing delivery vehicles. Formerly known as Endcadia, Verve Therapeutics rebranded in September 2019 to reflect its commitment to addressing heart disease through advanced genetic solutions.

Kymera Therapeutics

Series C in 2020
Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, established in 2015. It specializes in targeted protein degradation, developing innovative small molecule therapeutics that selectively degrade proteins associated with various diseases by utilizing the body's natural protein degradation mechanisms. The company is advancing several therapeutic programs, including the IRAK4 program aimed at treating immunology-inflammation diseases such as hidradenitis suppurativa, atopic dermatitis, and rheumatoid arthritis. Additionally, it is developing the IRAKIMiD program for MYD88-mutated diffuse large B cell lymphoma and the STAT3 program for hematologic malignancies and solid tumors, as well as autoimmune diseases. Through its proprietary predictive modeling platform, Kymera Therapeutics seeks to accelerate drug discovery and address previously untreatable conditions.

Generation Bio

Series C in 2020
Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing gene therapies aimed at treating both rare and prevalent diseases. Established in 2016, the company focuses on conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs an innovative non-viral platform that includes a proprietary high-capacity DNA construct known as closed-ended DNA (ceDNA), a cell-targeted lipid nanoparticle delivery system (ctLNP), and a scalable, capsid-free manufacturing process. This approach allows for the targeted delivery of genetic materials, including large and multiple genes across various tissues, and enables redosable treatments tailored to individual patient needs. By enhancing access to therapies for rare diseases and addressing more common conditions, Generation Bio aims to provide durable and sustainable treatment options for patients throughout their lives.

Aligos Therapeutics

Series B in 2020
Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company established in 2018 and based in South San Francisco, California. The company specializes in developing novel therapeutics aimed at addressing unmet medical needs in viral and liver diseases. Its lead candidate, ALG-010133, is a synthetic oligonucleotide currently undergoing Phase I clinical trials for chronic hepatitis B (CHB). In addition to this, Aligos is advancing several other drug candidates, including ALG-000184, a capsid assembly modulator for CHB; ALG-020572, an oligonucleotide for CHB; ALG-125097, an siRNA drug candidate for CHB; and ALG-055009, a small molecule THR-ß agonist targeting non-alcoholic steatohepatitis (NASH). The company leverages its extensive expertise in liver disease and viral hepatitis to develop targeted antiviral therapies, positioning itself as a leader in this therapeutic area.

FORMA Therapeutics

Series D in 2019
Forma Therapeutics Holdings, Inc., a clinical-stage biopharmaceutical company, engages in the development and commercialization of novel therapeutics for treatment of rare hematologic diseases and cancers. Its core product candidates for development include FT-4202, which is Phase 1 trial for the treatment of sickle cell disease and other hemoglobinopathies; and FT-7051 for the treatment of metastatic castration-resistant prostate cancer. The company is also developing FT-2102, an oral selective small molecule investigational agent that is designed to bind to and inhibit mutated IDH1 enzymes, which is being evaluated in a Phase 2 trial for relapsed/refractory AML, as well as an exploratory Phase 1 trial for glioma; and FT-4101 and FT-8225, which are selective fatty acid synthase inhibitors. The company was founded in 2007 and is headquartered in Watertown, Massachusetts.

Black Diamond Therapeutics

Series C in 2019
Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule, tumor-agnostic therapies. The company's lead product candidate, BDTX-189, is designed to inhibit specific mutations in ErbB kinases, including the epidermal growth factor receptor. Additionally, Black Diamond is advancing a pipeline of allosteric-EGFR mutation inhibitors and other early-stage programs targeting allosteric mutations in kinases associated with cancer and rare genetic diseases. The company employs a technology platform known as Mutation-Allostery-Pharmacology, which aims to address undrugged mutations in patients with genetically defined cancers. Founded in 2014 and based in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its rebranding in 2018.

Satsuma Pharmaceuticals

Series B in 2019
Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraines. The company's primary product candidate, STS101, is a drug-device combination featuring a proprietary dry-powder formulation of dihydroergotamine mesylate. This medication is designed for self-administration using a pre-filled, single-use nasal delivery device. Currently, STS101 is undergoing Phase III clinical trials, marking a significant step in its development process. Founded in 2016 and based in South San Francisco, California, Satsuma Pharmaceuticals aims to provide effective solutions for individuals suffering from acute migraine attacks.

Neon Therapeutics

Series B in 2017
Neon Therapeutics is a clinical-stage biopharmaceutical company. It is focused in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. The company's pipeline products are NEO-PV-01, NEO-PTC-01, and NEO-SV-01.

Nightstar Therapeutics

Series C in 2017
Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

Neon Therapeutics

Series B in 2017
Neon Therapeutics is a clinical-stage biopharmaceutical company. It is focused in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. The company's pipeline products are NEO-PV-01, NEO-PTC-01, and NEO-SV-01.

Mersana Therapeutics

Series C in 2016
Mersana Therapeutics employs its biodegradable polymer platform to create new and better medicines. They are advancing their own clinical-stage pipeline of novel compounds with the potential to address multiple oncology indications. It was founded in 2005 and headquartered in Cambridge, Massachusetts.

CytomX Therapeutics

Series D in 2015
CytomX Therapeutics is an early stage, privately-funded biotechnology company developing Probodiesâ„¢, proteolytically-activated antibodies. Probodies, by their ability to site-direct the activity of antibodies, will result in an improved therapeutic index for validated targets as well enable the drugging of targets with broad tissue expression. Their mission, alone and with the help of partners, is to provide patients with more effective and less toxic therapies for severe illnesses such as cancer and inflammatory diseases.

UNUM Therapeutics

Series B in 2015
Unum Therapeutics is a biopharmaceutical company focused on developing immunotherapy products for cancer treatment, utilizing proprietary T-cell engineering technology. The company’s lead programs, based on its Antibody-Coupled T-cell Receptor (ACTR) technology, are in Phase I clinical trials. These include ACTR707 and ACTR087, both being tested in combination with rituximab for relapsed or refractory CD20+ non-Hodgkin lymphoma, as well as ACTR087 combined with SEA-BCMA for relapsed multiple myeloma. Additionally, ACTR707 is being evaluated with trastuzumab for HER2+ solid tumors. Unum Therapeutics is also exploring BOXR1030, a pre-clinical candidate targeting glypican-3 to enhance T cell functionality in the solid tumor microenvironment. Founded in 2014 and headquartered in Cambridge, Massachusetts, Unum aims to activate the body’s immune system to effectively combat cancer through its innovative therapies.

Jounce Therapeutics

Series B in 2015
Jounce Therapeutics is a clinical-stage immunotherapy company focused on developing innovative cancer treatments that harness the immune system to target and eliminate cancer cells. The company's lead product, vopratelimab, is a monoclonal antibody currently in Phase II trials for patients with non-small cell lung cancer and urothelial cancer, particularly those who have not responded to PD-1/PD-L1 inhibitors. Additionally, Jounce is advancing several other therapeutic candidates, including JTX-4014, an anti-PD-1 antibody intended for combination therapy, and JTX-1811, which aims to selectively deplete T regulatory cells in the tumor microenvironment. The company also develops JTX-8064, an antibody targeting a receptor on macrophages. Founded in 2013 by experts in immunobiology and cancer research, Jounce Therapeutics is headquartered in Cambridge, Massachusetts, and is committed to improving patient outcomes through its proprietary immunotherapy platform.

Voyager Therapeutics

Series B in 2015
Voyager Therapeutics is a gene therapy company developing life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS). Voyager is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and investment in vector optimization and engineering, dosing techniques, as well as process development and production. The company’s initial pipeline is focused on CNS diseases in dire need of effective new therapies, including Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia. Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience, Voyager Therapeutics was launched in 2014 with funding from leading life sciences investor Third Rock Ventures and is headquartered in Cambridge, Mass.

Global Blood Therapeutics

Series B in 2015
GBT is a clinical-stage biopharmaceutical company that focuses on discovering, developing, and delivering innovative treatments that provide hope to underserved patient communities. GBT is developing its product candidate, voxelotor, as an oral, once-daily therapy for sickle cell disease. It was founded in 2012 and headquartered in San Francisco, California.

Moderna

Series E in 2015
Moderna, Inc. is a biotechnology company based in Cambridge, Massachusetts, founded in 2010. It specializes in the development of therapeutics and vaccines utilizing messenger RNA technology, which instructs cells to produce proteins essential for various biological functions. The company's extensive pipeline includes 40 mRNA development candidates targeting a range of therapeutic areas such as infectious diseases, oncology, cardiovascular diseases, and rare genetic disorders. Moderna gained significant recognition with its COVID-19 vaccine, which was authorized for use in the United States in December 2020. The company maintains strategic partnerships with organizations such as AstraZeneca, Merck & Co., and the Bill & Melinda Gates Foundation, enhancing its research capabilities and manufacturing processes. Additionally, it has research collaborations with esteemed institutions like Harvard University.

Nivalis Therapeutics

Venture Round in 2014
Nivalis Therapeutics is developing a novel class of disease-modifying therapies that are designed to preserve intracellular GSNO (S-nitrosoglutathione), an endogenous molecule with cell signaling effects that are implicated in the pathophysiology of cystic fibrosis (CF).The Company’s lead candidate, N91115 initially targets patients with the F508del mutation, the most common disease-causing mutation in CF. Nivalis Therapeutics is dedicated to making a difference in the lives of patients with cystic fibrosis and their families. The company was founded in 2007 and is headquartered in Boulder, Colorado, United States.

Blueprint Medicines

Series C in 2014
Blueprint Medicines Corporation focuses on developing small molecule kinase inhibitors aimed at treating genomic drivers in cancers, rare diseases, and enhancing cancer immunotherapy. The company is advancing several key drug candidates, including avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis and other mast cell disorders, and fisogatinib for advanced hepatocellular carcinoma, currently in Phase I clinical trials. Additionally, pralsetinib targets RET-altered non-small cell lung cancer and other solid tumors, while BLU-782 is being investigated for fibrodysplasia ossificans progressive. Founded in 2008 and headquartered in Cambridge, Massachusetts, Blueprint Medicines emphasizes the development of personalized cancer therapies by leveraging insights into cancer genomics and utilizing a proprietary chemical library to identify new therapeutic compounds. The company collaborates with various partners, including Clementia Pharmaceuticals and Genentech, to enhance its research and development efforts.

Adaptimmune

Series A in 2014
Adaptimmune Therapeutics is a clinical-stage biopharmaceutical company based in Abingdon, United Kingdom, specializing in innovative T-cell therapies for cancer and other diseases. The company's proprietary peptide enhanced affinity receptor (SPEAR) T-cell platform allows for the identification of specific cancer targets, enabling the development of therapies aimed at solid tumors. Adaptimmune is currently advancing several products through clinical trials, including ADP-A2M4, which targets various solid tumors such as melanoma and non-small cell lung cancer; ADP-A2AFP for hepatocellular carcinoma; and ADP-A2M10 for multiple cancer indications. The company has formed strategic collaborations to enhance its research and development efforts, including partnerships with GSK for NY-ESO T-cell therapy and Noile-Immune Biotech for combining SPEAR T-cells with PRIME technology. Adaptimmune was founded in 2008 and is committed to delivering novel cell therapies to improve patient outcomes.

Civitas Therapeutics

Series C in 2014
Civitas Therapeutics is a biopharmaceutical company dedicated to developing therapies for pulmonary delivery. The company focuses on creating innovative treatments for central nervous system and respiratory disorders, with a particular emphasis on conditions such as multiple sclerosis and spinal cord injuries. Civitas Therapeutics aims to improve the health and quality of life for patients across the United States through its specialized drug delivery systems.

Spark Therapeutics

Series B in 2014
Spark Therapeutics, Inc. is dedicated to developing gene therapy products aimed at treating debilitating genetic diseases. The company is known for LUXTURNA, a treatment for patients with biallelic RPE65 mutation-associated retinal dystrophy, and has an extensive pipeline that includes candidates like SPK-8011 and SPK-8016 for hemophilia, SPK-7001 for choroideremia, and SPK-9001 for hemophilia B. Additionally, Spark is exploring liver-directed gene therapies such as SPK-3006 for Pompe disease and has programs targeting neurodegenerative diseases, including Huntington's disease and a form of Batten disease. Spark Therapeutics also engages in collaborations, notably with Pfizer for the SPK-FIX program for hemophilia B, and has a licensing agreement with Novartis for the commercialization of LUXTURNA outside the United States. Established in 2013 and based in Philadelphia, Spark Therapeutics operates as a subsidiary of Roche Holding AG and leverages a proprietary manufacturing platform to support its clinical studies across various therapeutic areas.