DISEASE CATEGORY: Cancer INDICATION: Ovarian cancer; melanoma; colorectal cancer CAR T cells targeting folate receptor FOLR2 could treat ovarian cancer, melanoma and colorectal cancer. Among patients with stage III or IV ovarian cancer, high FOLR2...

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Obesity; liver disease Reducing levels of eIF4E or lowering the RNA cap-binding protein’s activity by blocking its phosphorylation could treat obesity...

DISEASE CATEGORY: Cancer INDICATION: Acute lymphoblastic leukemia (ALL) A CDK12-targeted degrader, designed by a Dana-Farber Cancer Institute and Harvard Medical School team co-led by Nathanael Gray, could treat T cell ALL. The degrader’s targeting moiety was derived...

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Contraception Triptonide, a compound purified from Tripterygium Wilfordii Hook F, could serve as a non-hormonal male contraceptive. In vitro assays identified JUP as triptonide’s most likely target. In mice and cynomolgus monkeys, oral triptonide led to...

DISEASE CATEGORY: Cancer INDICATION: Melanoma; lung cancer A Yale University and Memorial Sloan Kettering Cancer Center team led by Arvinas Inc. (NASDAQ:ARVN) founder Craig Crews created a PROTAC based on Zelboraf vemurafenib that could treat BRAF...

DISEASE CATEGORY: Autoimmune INDICATION: Arthritis; rheumatoid arthritis (RA) Inhibiting the endocytic protein SH3GL1 could treat autoimmune arthritis, including RA. CD4+ T cells from eight RA patients had higher mRNA levels of SH3GL1 than cells from...

DISEASE CATEGORY: Infectious disease INDICATION: Coronavirus; influenza CRISPR-Cas13a knockdown of the nucleocapsid protein of SARS-CoV-2 or influenza’s viral RNA polymerase could treat COVID-19 or flu, respectively. In monkey kidney cells infected with SARS-CoV-2, Cas13a targeting of the...

DISEASE CATEGORY: Neurology INDICATION: Alzheimer's disease (AD); Huntington disease (HD); Parkinson's disease (PD) Inhibiting STS, which removes sulfates from inactive steroid hormones, could treat Alzheimer’s, Huntington and Parkinson’s diseases and other aging-related indications. In a  Caenorhabditis...

DISEASE CATEGORY: Infectious disease INDICATION: Sepsis Disrupting the interaction between the immunoglobulin-like cell surface receptor RAGE and the adaptor protein LCP2 could treat sepsis. Immunoprecipitation assays revealed LCP2’s sterile α motif domain binds RAGE’s cytosolic tail. In...

DISEASE CATEGORY: Ophthalmic disease INDICATION: Age-related macular degeneration (AMD) A CRISPR-Cas9-based lentiviral vector gene therapy targeting VEGF-A could prevent wet AMD. In a mouse model of laser-induced wet AMD, pretreatment with the gene therapy injected subretinally...