Atlas Venture

Trace Neuroscience is a biopharmaceutical company dedicated to advancing genetic medicine for patients with neurodegenerative diseases. It specializes in developing targeted antisense oligonucleotide therapies, with its lead product aimed at restoring UNC13A protein function in individuals with Amyotrophic Lateral Sclerosis (ALS) caused by TDP-43 mislocalization. This therapy corrects UNC13A mRNA splicing, potentially improving muscle function and extending survival for these patients.

Judo Bio is a biotechnology company focused on developing precision therapeutics that target specific cell populations to treat genetic diseases. Utilizing an innovative receptor targeting approach, Judo Bio is building a pipeline of treatments designed to address both common and rare diseases. Currently, the company is operating in stealth mode, indicating a period of research and development prior to a public launch or disclosure of its initiatives.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

GRO Biosciences is a biotechnology company focused on developing advanced protein therapeutics to address various health conditions, including diabetes, growth disorders, and autoimmunity. The company employs innovative technologies in computational protein design and synthetic biology, which enable it to create genomically recoded strains of bacteria capable of incorporating non-standard amino acids into proteins. This approach enhances the properties of the proteins, resulting in increased potency, stability, and targeted delivery. Through its technology platform, GRO Biosciences aims to provide improved treatment options across multiple disease classes, contributing to advancements in medical therapeutics.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Judo Bio is a biotechnology company focused on developing precision therapeutics that target specific cell populations to treat genetic diseases. Utilizing an innovative receptor targeting approach, Judo Bio is building a pipeline of treatments designed to address both common and rare diseases. Currently, the company is operating in stealth mode, indicating a period of research and development prior to a public launch or disclosure of its initiatives.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Nvelop Therapeutics is a biotechnology company dedicated to developing advanced genetic medicines aimed at treating severe genetic diseases. The company specializes in creating innovative, programmable delivery systems that facilitate efficient in vivo gene editing and the delivery of therapeutic cargo. By integrating epigenetic editing technologies with next-generation delivery methods, Nvelop Therapeutics addresses significant unmet medical needs. Its focus is on delivering a range of therapeutic payloads, initially prioritizing gene editing within therapeutically relevant cells and tissues, thereby enhancing the potential for effective treatment options for patients suffering from serious genetic conditions.

AAVantgarde Bio is a biotechnology company focused on developing gene therapies specifically for inherited retinal disorders. The company utilizes proprietary Adeno-Associated Viral (AAV) vector platforms to overcome the limitations of traditional AAV vectors, particularly in terms of cargo capacity. This innovative approach allows for the delivery of large genes to both ocular and non-ocular tissues, facilitating effective in vivo treatment options for patients suffering from these conditions. Through its advanced technology, AAVantgarde Bio aims to enhance therapeutic outcomes and improve the quality of life for individuals affected by genetic eye diseases.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Rectify Pharmaceuticals is focused on creating disease-modifying precision therapies aimed at addressing the root causes of serious genetic diseases. The company specializes in developing positive functional modulators (PFMs) that restore the function of ABC transporters, which are critical for cellular processes. By targeting these underlying mechanisms, Rectify aims to provide innovative treatment solutions that improve the health outcomes of patients suffering from various genetic conditions. Their research and development efforts encompass multiple therapeutic areas, reflecting a commitment to advancing precision medicine in the field of genetic disorders.

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

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Dyne Therapeutics, Inc. is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral muscular dystrophy, as well as rare skeletal, cardiac, and metabolic muscle diseases. Utilizing its proprietary FORCE platform, Dyne Therapeutics delivers nucleic acids and other therapeutic molecules to muscle tissues with high precision, aiming to restore muscle strength and improve patients' quality of life. The company is currently advancing its product candidates, DYNE-101 and DYNE-251, which are in phase 1/2 clinical trials.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

Generation Bio Co. is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies aimed at treating both rare and prevalent diseases. Founded in 2016 and formerly known as Torus Therapeutics, the company focuses on creating durable and redosable treatments for conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs a proprietary platform that utilizes closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP) to achieve targeted delivery of genetic materials across various tissues. This approach is designed to enhance treatment efficacy while expanding access to therapies for rare diseases and addressing more common conditions through scalable manufacturing processes.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Triplet Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies for triplet repeat expansion disorders, such as Huntington's disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these conditions at their source by utilizing a unique approach that involves a single oligonucleotide targeting the DNA Damage Response pathway. This strategy is designed to provide effective treatments for multiple repeat expansion disorders, enabling healthcare professionals to offer advanced biotherapeutic options to their patients. Through its research and development efforts, Triplet Therapeutics is committed to transforming the landscape of treatment for these challenging genetic disorders.

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

Dyne Therapeutics, Inc. is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral muscular dystrophy, as well as rare skeletal, cardiac, and metabolic muscle diseases. Utilizing its proprietary FORCE platform, Dyne Therapeutics delivers nucleic acids and other therapeutic molecules to muscle tissues with high precision, aiming to restore muscle strength and improve patients' quality of life. The company is currently advancing its product candidates, DYNE-101 and DYNE-251, which are in phase 1/2 clinical trials.

Triplet Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies for triplet repeat expansion disorders, such as Huntington's disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these conditions at their source by utilizing a unique approach that involves a single oligonucleotide targeting the DNA Damage Response pathway. This strategy is designed to provide effective treatments for multiple repeat expansion disorders, enabling healthcare professionals to offer advanced biotherapeutic options to their patients. Through its research and development efforts, Triplet Therapeutics is committed to transforming the landscape of treatment for these challenging genetic disorders.

AVROBIO, Inc. is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing ex vivo lentiviral-based therapies aimed at treating rare diseases with a single-dose regimen. The company utilizes hematopoietic stem cells collected from patients, which are modified using a lentiviral vector to introduce functional copies of defective genes associated with specific diseases. AVROBIO's lead product candidate, AVR-RD-01, is currently undergoing Phase II clinical trials for Fabry disease, while AVR-RD-02 and AVR-RD-04 have completed pre-clinical trials for type 1 Gaucher disease and cystinosis, respectively. Additionally, AVR-RD-03 is in preclinical development for Pompe disease. The company has also established a research collaboration with Magenta Therapeutics to explore targeted antibody-drug conjugates as conditioning regimens for its lentiviral gene therapies. Founded in 2015, AVROBIO aims to accelerate advancements in cell and gene therapies to address serious and debilitating conditions.

Generation Bio Co. is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies aimed at treating both rare and prevalent diseases. Founded in 2016 and formerly known as Torus Therapeutics, the company focuses on creating durable and redosable treatments for conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs a proprietary platform that utilizes closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP) to achieve targeted delivery of genetic materials across various tissues. This approach is designed to enhance treatment efficacy while expanding access to therapies for rare diseases and addressing more common conditions through scalable manufacturing processes.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

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.

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

AVROBIO, Inc. is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing ex vivo lentiviral-based therapies aimed at treating rare diseases with a single-dose regimen. The company utilizes hematopoietic stem cells collected from patients, which are modified using a lentiviral vector to introduce functional copies of defective genes associated with specific diseases. AVROBIO's lead product candidate, AVR-RD-01, is currently undergoing Phase II clinical trials for Fabry disease, while AVR-RD-02 and AVR-RD-04 have completed pre-clinical trials for type 1 Gaucher disease and cystinosis, respectively. Additionally, AVR-RD-03 is in preclinical development for Pompe disease. The company has also established a research collaboration with Magenta Therapeutics to explore targeted antibody-drug conjugates as conditioning regimens for its lentiviral gene therapies. Founded in 2015, AVROBIO aims to accelerate advancements in cell and gene therapies to address serious and debilitating conditions.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

AVROBIO, Inc. is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing ex vivo lentiviral-based therapies aimed at treating rare diseases with a single-dose regimen. The company utilizes hematopoietic stem cells collected from patients, which are modified using a lentiviral vector to introduce functional copies of defective genes associated with specific diseases. AVROBIO's lead product candidate, AVR-RD-01, is currently undergoing Phase II clinical trials for Fabry disease, while AVR-RD-02 and AVR-RD-04 have completed pre-clinical trials for type 1 Gaucher disease and cystinosis, respectively. Additionally, AVR-RD-03 is in preclinical development for Pompe disease. The company has also established a research collaboration with Magenta Therapeutics to explore targeted antibody-drug conjugates as conditioning regimens for its lentiviral gene therapies. Founded in 2015, AVROBIO aims to accelerate advancements in cell and gene therapies to address serious and debilitating conditions.

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.

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

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

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

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.

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.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

On-Q-ity is an innovative diagnostic company that is focused on providing physicians with the tools they need to make the best decision when treating individual cancer patients. There platform will enable better decision-making across multiple cancers: improving the quality of life for patients and ultimately increasing survival. Products currently in development are based on two core technologies: 1) DNA repair biomarkers that predict treatment response and 2) Microfluidic chip technology that enables capture, enumeration and characterization of circulating tumor cells (CTCs) in the bloodstream.

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

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

On-Q-ity is an innovative diagnostic company that is focused on providing physicians with the tools they need to make the best decision when treating individual cancer patients. There platform will enable better decision-making across multiple cancers: improving the quality of life for patients and ultimately increasing survival. Products currently in development are based on two core technologies: 1) DNA repair biomarkers that predict treatment response and 2) Microfluidic chip technology that enables capture, enumeration and characterization of circulating tumor cells (CTCs) in the bloodstream.

Helicos BioSciences Corporation (Helicos) is a life sciences company, which has developed technology focused on the research, drug discovery and clinical diagnostics markets. Its True Single Molecule Sequencing (tSMS) technology enables rapid analysis of large quantities of genetic material by directly sequencing single molecules of deoxyribonucleic acid (DNA) or single DNA copies of ribonucleic acid (RNA) (cDNA) and its approach of direct sequencing of RNA. Its Helicos Genetic Analysis Platform is designed to obtain sequencing information by repetitively performing a cycle of biochemical reactions on individual DNA or RNA molecules and imaging the results after each cycle. The platform consists of an instrument called the HeliScope Single Molecule Sequencer, an image analysis computer tower called the HeliScope Analysis Engine, associated reagents, which are chemicals used in the sequencing process and disposable supplies.

Adnexus, a Bristol-Myers Squibb R&D Company, specializes in the discovery and development of Adnectins, a unique class of targeted biologics. These proteins are engineered to block or stimulate therapeutic targets to combat various diseases, addressing significant medical needs in oncology, immunology, and cardiovascular health. Utilizing a proprietary protein engineering system known as PROfusion™, Adnexus can generate a vast library of over 10 trillion potential Adnectins. This technology enhances the efficiency of identifying candidates with desirable therapeutic properties. Among its product candidates is Angiocept/CT-322, an anti-angiogenesis agent aimed at treating cancer by inhibiting the VEGFR-2 pathway. Since its acquisition by Bristol-Myers Squibb in 2007, Adnexus has further advanced its capabilities in developing innovative medicines to improve patient outcomes.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Adnexus, a Bristol-Myers Squibb R&D Company, specializes in the discovery and development of Adnectins, a unique class of targeted biologics. These proteins are engineered to block or stimulate therapeutic targets to combat various diseases, addressing significant medical needs in oncology, immunology, and cardiovascular health. Utilizing a proprietary protein engineering system known as PROfusion™, Adnexus can generate a vast library of over 10 trillion potential Adnectins. This technology enhances the efficiency of identifying candidates with desirable therapeutic properties. Among its product candidates is Angiocept/CT-322, an anti-angiogenesis agent aimed at treating cancer by inhibiting the VEGFR-2 pathway. Since its acquisition by Bristol-Myers Squibb in 2007, Adnexus has further advanced its capabilities in developing innovative medicines to improve patient outcomes.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

Adnexus, a Bristol-Myers Squibb R&D Company, specializes in the discovery and development of Adnectins, a unique class of targeted biologics. These proteins are engineered to block or stimulate therapeutic targets to combat various diseases, addressing significant medical needs in oncology, immunology, and cardiovascular health. Utilizing a proprietary protein engineering system known as PROfusion™, Adnexus can generate a vast library of over 10 trillion potential Adnectins. This technology enhances the efficiency of identifying candidates with desirable therapeutic properties. Among its product candidates is Angiocept/CT-322, an anti-angiogenesis agent aimed at treating cancer by inhibiting the VEGFR-2 pathway. Since its acquisition by Bristol-Myers Squibb in 2007, Adnexus has further advanced its capabilities in developing innovative medicines to improve patient outcomes.

Variagenics is a biotechnology company that focuses on the development and commercialization of proprietary gene variance detection technology. This technology is designed to enhance and streamline the drug development process. In addition to its core activities in gene variance detection, Variagenics is involved in the manufacturing and development of molecular diagnostic products. The company serves clients primarily in the United States, offering innovative solutions aimed at improving drug discovery and diagnostic accuracy. Through its advancements in genetic diagnostics, Variagenics plays a significant role in the evolving landscape of personalized medicine.

DeCODE Genetics is a biopharmaceutical company based in Reykjavik, Iceland, founded in 1996. It focuses on analyzing the human genome to identify genetic variants associated with common diseases. The company utilizes population studies to uncover key genetic risk factors for various conditions, including cardiovascular disease and cancer. DeCODE Genetics develops diagnostic tests and technologies aimed at improving the treatment, diagnosis, and prevention of these diseases. By leveraging its expertise in chemistry and structural biology, the company also supports clients in the healthcare sector with DNA-based reference laboratory tests and consumer genome analysis services, which help assess individual risk for common diseases.