EcoR1 Capital

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs targeting G protein-coupled receptors (GPCRs). By utilizing its proprietary technology platform, GEODe, Tectonic aims to create therapeutic proteins and antibodies that effectively modulate GPCR activity. The company seeks to address the complexities associated with GPCR-targeted drug discovery, focusing on developing biologic medicines that have the potential to alter disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic is committed to advancing the therapeutic utility of some of the most challenging receptors in this class.

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs targeting G protein-coupled receptors (GPCRs). By utilizing its proprietary technology platform, GEODe, Tectonic aims to create therapeutic proteins and antibodies that effectively modulate GPCR activity. The company seeks to address the complexities associated with GPCR-targeted drug discovery, focusing on developing biologic medicines that have the potential to alter disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic is committed to advancing the therapeutic utility of some of the most challenging receptors in this class.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to create targeted treatments that address critical cytokine signaling pathways involved in these diseases. By integrating insights from disease mechanisms, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to design precise therapeutics that meet significant unmet medical needs for patients suffering from hematologic malignancies.

CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.

At Kura Oncology, They are committed to realizing the promise of precision medicines for cancer. The genomics revolution is transforming how they treat cancer. They now understand that how a patient responds to treatment depends in part on the genetic makeup of the cancer and, importantly, we have the knowledge and tools to create targeted treatments and companion diagnostics to identify those patients most likely to benefit. This new era offers the potential for innovative treatments that are safer and more effective for patients with particular cancers. They are leveraging our insights into cancer genetics as well as our core strength of translating novel science into life-saving medicines to advance a pipeline of precision medicines. Their development programs target cancers with high unmet need, including lung, colorectal, thyroid and pancreatic cancers as well as blood cancers such as lymphoma and leukemias.

Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.

ReCode Therapeutics is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

Actio Biosciences focuses on the development of precision medicines tailored for homogeneous rare patient populations. The company is engaged in creating a pipeline of innovative drugs aimed at treating rare diseases, with the potential to extend their application to more common illnesses. By utilizing biotechnology to modulate the activity of proteins linked to rare diseases, Actio Biosciences aims to enhance the likelihood of clinical success and deliver effective treatments for conditions that are often difficult to cure. Their commitment to addressing both rare and common diseases positions them to make a significant impact in the field of medicine.

Paratus Sciences is a biotechnology company focused on enhancing human health and health security through the study of bat biology. By exploring the unique aspects of the bat genome, the company aims to identify and develop therapeutics for a variety of diseases. Paratus Sciences seeks to unlock the secrets of bat biology to facilitate innovative treatments, ultimately helping patients combat the health challenges they encounter.

Abeona Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing gene and cell therapies for severe, life-threatening rare genetic diseases. The company's leading programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, and ABO-102 and ABO-101, which are AAV-based gene therapies targeting Sanfilippo syndrome types A and B, respectively. Additional therapies under development include ABO-201 and ABO-202 for Batten disease, ABO-401 for cystic fibrosis, and ABO-5OX for genetic eye disorders. Abeona also operates the AIM Vector Platform, which focuses on advancing gene therapy delivery methods using next-generation AAV capsids. The company is engaged in a licensing agreement with REGENXBIO for gene therapies addressing multiple genetic disorders. Founded in 1974 and headquartered in New York, Abeona has a fully operational manufacturing facility that supports its preclinical and clinical research efforts.

22seventy bio is an immuno-oncology cell therapy company that discovers and develops cancer therapies. The company combines a deep knowledge of cancer cell biology and genetics with an understanding of the immune response to cancer to advance its pipeline. It was established in Cambridge, Massachusetts in 2021.

ReCode Therapeutics is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to create targeted treatments that address critical cytokine signaling pathways involved in these diseases. By integrating insights from disease mechanisms, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to design precise therapeutics that meet significant unmet medical needs for patients suffering from hematologic malignancies.

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs targeting G protein-coupled receptors (GPCRs). By utilizing its proprietary technology platform, GEODe, Tectonic aims to create therapeutic proteins and antibodies that effectively modulate GPCR activity. The company seeks to address the complexities associated with GPCR-targeted drug discovery, focusing on developing biologic medicines that have the potential to alter disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic is committed to advancing the therapeutic utility of some of the most challenging receptors in this class.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Syros Pharmaceuticals is a life sciences company that is focused on treating disease by mapping gene regulatory circuits and modulating the factors that regulate gene expression. Syros has pioneered world-leading gene control research and drug discovery capabilities with a proven ability to integrate disease biology and genomic data--a proficiency that is not well represented in pharmaceutical R&D. Central to the Syros approach is a proprietary platform of carefully integrated assay technologies, bioinformatics, and biologic insights developed by members of Syros' senior leadership. While this scientific approach has applications in many therapeutic areas, Syros has demonstrated success initially in oncology, where it may help address numerous unmet medical needs.

Neogene Therapeutics, Inc. is a biotechnology company based in New York, founded in 2018, that focuses on developing T cell therapies for cancer treatment. The company specializes in creating personalized engineered T cells that target neo-antigens, which are mutated proteins present in cancer cells due to DNA mutations. By leveraging advanced technologies, Neogene isolates T cell receptor (TCR) genes specific to these neo-antigens from tumor biopsies routinely collected from patients. This process utilizes high-sensitivity DNA sequencing and genetic screening to identify TCRs that can effectively recognize and attack cancer cells. Neogene's innovative approach aims to enhance the precision and efficacy of cancer therapies, offering improved treatment options for patients facing a broad spectrum of cancers.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

Intellia Therapeutics is a genome editing company headquartered in Cambridge, Massachusetts, that specializes in developing innovative therapeutics using the CRISPR/Cas9 technology. The company focuses on in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia's ex vivo pipeline includes engineered cell therapies aimed at treating various oncological and autoimmune diseases. The company has entered into collaborations with notable organizations, including Novartis and Regeneron Pharmaceuticals, to advance its research and development efforts. Founded in 2014, Intellia Therapeutics is committed to addressing severe and life-threatening diseases through its comprehensive intellectual property platform and a broad range of clinical development programs.

At Kura Oncology, They are committed to realizing the promise of precision medicines for cancer. The genomics revolution is transforming how they treat cancer. They now understand that how a patient responds to treatment depends in part on the genetic makeup of the cancer and, importantly, we have the knowledge and tools to create targeted treatments and companion diagnostics to identify those patients most likely to benefit. This new era offers the potential for innovative treatments that are safer and more effective for patients with particular cancers. They are leveraging our insights into cancer genetics as well as our core strength of translating novel science into life-saving medicines to advance a pipeline of precision medicines. Their development programs target cancers with high unmet need, including lung, colorectal, thyroid and pancreatic cancers as well as blood cancers such as lymphoma and leukemias.