Mission BioCapital

Arkuda Therapeutics is a biotechnology company leveraging new insights into progranulin and lysosomal biology to develop medicines to change the trajectory of neurodegenerative disease. Arkuda’s lead compounds aim to correct progranulin deficiency and lysosomal dysfunction in GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the GRN gene.

Tune Therapeutics is focused on developing innovative cell and gene therapies that utilize epigenetic programming to address serious diseases. By harnessing the potential of the epigenome, the company aims to create new therapeutic pathways that modify biological functions without changing the underlying DNA sequence. This approach seeks to provide effective treatments for complex and widespread diseases, positioning Tune Therapeutics at the forefront of advancements in epigenetic medicine.

Shape Therapeutics, Inc. is a biotechnology company based in Seattle, Washington, focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic diseases. Founded in 2018, the company has developed RNAfix technology, which utilizes the natural cellular machinery of the human body to edit RNA. This approach significantly reduces the risks of immunogenicity, cellular toxicity, and off-target DNA editing that are often associated with traditional gene-editing methods, such as CRISPR. Shape Therapeutics aims to create next-generation gene therapies that provide effective and safer treatment options for patients with challenging medical conditions.

Shape Therapeutics, Inc. is a biotechnology company based in Seattle, Washington, focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic diseases. Founded in 2018, the company has developed RNAfix technology, which utilizes the natural cellular machinery of the human body to edit RNA. This approach significantly reduces the risks of immunogenicity, cellular toxicity, and off-target DNA editing that are often associated with traditional gene-editing methods, such as CRISPR. Shape Therapeutics aims to create next-generation gene therapies that provide effective and safer treatment options for patients with challenging medical conditions.

Glycomine, Inc. is a biotechnology company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.

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.

Caribou Biosciences, Inc. is a biotechnology company based in Berkeley, California, specializing in cellular engineering and genome analysis through CRISPR-Cas9 technology. Founded in 2011, the company provides a gene editing platform that allows for targeted modifications in the genome. Its applications span various sectors, including human and animal therapeutics, agricultural biotechnology, and industrial biotechnology. Caribou's solutions facilitate the development of new disease models, enhance traits in plants, and support functional genomic screens. The company aims to advance both basic and applied biological research, leveraging its transformative capabilities to impact therapeutic development and bioproduction.

Arkuda Therapeutics is a biotechnology company leveraging new insights into progranulin and lysosomal biology to develop medicines to change the trajectory of neurodegenerative disease. Arkuda’s lead compounds aim to correct progranulin deficiency and lysosomal dysfunction in GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the GRN gene.

Shape Therapeutics, Inc. is a biotechnology company based in Seattle, Washington, focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic diseases. Founded in 2018, the company has developed RNAfix technology, which utilizes the natural cellular machinery of the human body to edit RNA. This approach significantly reduces the risks of immunogenicity, cellular toxicity, and off-target DNA editing that are often associated with traditional gene-editing methods, such as CRISPR. Shape Therapeutics aims to create next-generation gene therapies that provide effective and safer treatment options for patients with challenging medical conditions.

Shape Therapeutics, Inc. is a biotechnology company based in Seattle, Washington, focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic diseases. Founded in 2018, the company has developed RNAfix technology, which utilizes the natural cellular machinery of the human body to edit RNA. This approach significantly reduces the risks of immunogenicity, cellular toxicity, and off-target DNA editing that are often associated with traditional gene-editing methods, such as CRISPR. Shape Therapeutics aims to create next-generation gene therapies that provide effective and safer treatment options for patients with challenging medical conditions.

Glycomine, Inc. is a biotechnology company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.

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.

Sentieon, Inc. is a biotechnology company founded in 2014 and headquartered in Mountain View, California, specializing in cloud-based bioinformatics solutions for the life sciences and clinical markets. The company develops a suite of bioinformatics tools designed for the efficient processing of genomics data, focusing on high accuracy and computational efficiency. Key products include Sentieon DNAseq, which facilitates germline DNA analysis, and Sentieon TNseq, used for detecting somatic variants in tumor-normal samples. Sentieon's software solutions are characterized by their scalability, deployability, and upgradability, allowing them to adapt to various user needs. The company's advanced algorithms and robust software implementation ensure rapid turnaround times and consistent results, benefiting scientists, clinicians, and patients by enhancing precision in genomic data analysis.