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.

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.

Totient is an AI-driven biotechnology company that leverages tertiary lymphoid structures (TLSs) to identify novel tissue-specific antigens and develop matching high-affinity antibody therapeutics. It uses machine learning and immunoinformatics technology to pull critical insights from large, complex datasets, to quickly and accurately assemble the most potent antibody candidates. The unique applicability of the Totient platform allows the company to remain flexible and develop therapeutics for cancer, autoimmune disease, infections, and viruses. It was founded in 2017 and is based in Cambridge, Massachusetts.

Totient is an AI-driven biotechnology company that leverages tertiary lymphoid structures (TLSs) to identify novel tissue-specific antigens and develop matching high-affinity antibody therapeutics. It uses machine learning and immunoinformatics technology to pull critical insights from large, complex datasets, to quickly and accurately assemble the most potent antibody candidates. The unique applicability of the Totient platform allows the company to remain flexible and develop therapeutics for cancer, autoimmune disease, infections, and viruses. It was founded in 2017 and is based in Cambridge, Massachusetts.

QurAlis Corporation is a biotechnology company focused on discovering and developing precision therapeutics for amyotrophic lateral sclerosis (ALS) and other neurological diseases. Established in 2016 and based in Cambridge, Massachusetts, QurAlis aims to address the genetic underpinnings of ALS, leveraging its proprietary platforms and biomarkers to create targeted treatments. The company's pipeline includes innovative therapies designed to restore dysfunctional cellular processes, treat overactive neurons, and eliminate toxic proteins associated with the disease. By concentrating on genetically validated targets, QurAlis seeks to advance antisense oligonucleotides and small molecule programs that can effectively manage various subtypes of ALS, ultimately working to halt disease progression and improve patient outcomes. QurAlis operates as a subsidiary of Q-State Biosciences, Inc.

Pandion Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapeutics for autoimmune diseases. The company's lead product candidate, PT101, is an engineered variant of interleukin-2 currently undergoing Phase 1a clinical trials to address various autoimmune and inflammatory conditions. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and two bifunctional molecules, PT001 and PT002, designed to facilitate tissue-selective immunomodulation in the gastrointestinal tract. PT001 combines a PD-1 agonist with a tether module that binds to mucosal vascular addressing cell adhesion molecule (MAdCAM), while PT002 merges an IL-2 mutein effector with a MAdCAM-binding module. The company has also established a collaboration with Astellas Pharma to develop locally acting immunomodulators for pancreatic autoimmune diseases. Headquartered in Watertown, Massachusetts, Pandion Therapeutics aims to create effective and safe treatments to improve patient outcomes in the field of autoimmune disorders.

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.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. It specializes in developing and commercializing silk biomaterial technologies aimed at improving vaccine delivery and storage. The company has created the MIMIX microneedle platform, which facilitates transdermal delivery of vaccines and therapies, and the MATRIX formulation and drying platform, which enhances the stability of vaccines, therapeutics, and diagnostic products. By utilizing a silk-derived protein, Vaxess enables vaccines to be stored and transported without refrigeration, thereby reducing distribution costs and increasing access to essential healthcare products globally.

Comet Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, founded in 2018. It specializes in developing novel small-molecule treatments targeting various diseases, particularly orphan neurological disorders. The company's core innovation is its CoEnzyme metabolism platform, which aims to restore dysregulated CoEnzyme A metabolism. This therapeutic approach enables healthcare providers to address significant unmet medical needs in the treatment of these complex conditions.

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.

Nocion Therapeutics, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, established in 2017. The company focuses on developing novel therapeutics called "nocions," which are designed to treat conditions associated with neurogenic inflammation. These nocions specifically target activated sensory neurons, entering nociceptors through large-pore channels that open in response to stimulation. This mechanism allows for the selective blocking of sodium channels, providing targeted and sustained relief for serious conditions such as cough, itch, pain, and inflammation. By offering an alternative to traditional small molecule anesthetics, Nocion Therapeutics aims to deliver more effective treatment options for patients suffering from these debilitating symptoms.

Zymergen, Inc. is a biotechnology company that focuses on researching, developing, and manufacturing microbes for various industries, including agriculture, chemicals, materials, pharmaceuticals, electronics, and personal care. The company integrates automation, machine learning, and genomics to enhance its platform, which aids in optimizing microbial strains and improving the efficiency of production processes. By leveraging big data and artificial intelligence, Zymergen collaborates with clients to engineer novel molecules from microbes, thereby advancing product development. Founded in 2013 and headquartered in Emeryville, California, Zymergen also has offices in Boise, Idaho; Medford, Massachusetts; Seattle, Washington; and Tokyo, Japan, enabling it to serve a global clientele effectively.

QurAlis Corporation is a biotechnology company focused on discovering and developing precision therapeutics for amyotrophic lateral sclerosis (ALS) and other neurological diseases. Established in 2016 and based in Cambridge, Massachusetts, QurAlis aims to address the genetic underpinnings of ALS, leveraging its proprietary platforms and biomarkers to create targeted treatments. The company's pipeline includes innovative therapies designed to restore dysfunctional cellular processes, treat overactive neurons, and eliminate toxic proteins associated with the disease. By concentrating on genetically validated targets, QurAlis seeks to advance antisense oligonucleotides and small molecule programs that can effectively manage various subtypes of ALS, ultimately working to halt disease progression and improve patient outcomes. QurAlis operates as a subsidiary of Q-State Biosciences, Inc.