Focused on correcting disrupted brain physiology to treat neurodegenerative disease.
#Neuroscience
Dedicated to advancing in vivo cell reprogramming.
#Platform, delivery
Developing innovative immunotherapies to transform the tumor microenvironment and improve treatment options for cancer patients.
#Oncology
Dedicated to the research and development of novel therapies for the treatment of respiratory diseases.
#Respiratory
Developing immunoglobulin E (IgE) antibodies to treat cancer.
#Oncology
Developing small-molecule enzyme inhibitors that target distal binding pockets to reprogram enzyme activity with a focus on high-value oncology indications.
#Oncology, Platform
Pursuing the vast potential of small molecule medicines for RNA-mediated diseases.
#Platform, Muscular, Neuroscience
Inserting therapeutic genes precisely and durably into safe harbor sites in the human genome.
#Not specified
Developing breakthrough targeted medicines for precision oncology.
#Oncology
Focuses on the development of novel anti-cancer therapies by drugging molecular targets essential for cancer cells undergoing DNA replication stress.
#Oncology
Developing NMDA/TRPM4 interface inhibitors to stop neurodegeneration, restore function, and maintain brain health.
#Neuroscience
Engineers T cells to generate stable, scalable, IL2 and tissue targeted regulatory T cells better suited to address serious unmet medical needs for autoimmunity, alloimmunity, and autoinflammation.
#Immunology
Developing novel treatments for inflammation and autoimmunity.
#Immunology
Developing small-molecule covalent inhibitors targeting non-cysteine amino acids to address current challenges associated with many disease targets, including selectivity, druggability, and resistance.
#Platform
Focused on transformative treatments for cancers and autoimmune diseases.
#Cell therapy
Employs a single-cell genomics platform to dissect the biology of immune cells in human tumors and deliver meaningful clinical improvements.
#Immunology, Oncology
Focusing on targeting DNA damage repair pathways to effectively treat Huntington’s and other triplet repeat diseases.
#Neuroscience
Pioneering a new approach to fibrosis treatment.
#Fibrosis
Developing first-in-class therapeutics to protect and improve the vision of patients with retinal disease.
#Ophthalmology
Developing a first-in-class series of anti-cancer molecular glues that reactivate protein phosphatase 2A (PP2A).
#Oncology
Developing breakthrough medicines to exploit cancer’s vulnerabilities in gene expression.
#Oncology
Targeting cone cells for gene therapy.
#Gene therapy
Develops first-in-class therapeutics to target novel enzyme families activated under cellular stress conditions that contribute to disease.
#Immunology
Exploiting protein splicing to develop the next generation of gene therapies for the treatment of inherited eye diseases.
#Gene Therapy, Platform
Developing a new standard of care for patients suffering from severe diseases including cancer.
#Platform, Oncology
Developing an IgA therapeutic monoclonal antibody platform to activate neutrophils for the treatment of solid tumors.
#Oncology, Immunology
Focuses on the discovery and clinical development of gene therapy and protein replacement therapy for inherited metabolic liver diseases.
#Gene Therapy