Tagged: drug-discovery
30 results
Atavistik Bio
Atavistik Bio is a Cambridge, Massachusetts clinical-stage biotechnology company that discovers precision allosteric small-molecule medicines. Its proprietary AMPS (Atavistik Metabolite-Protein Screening) platform, paired with an AI-enabled discovery engine, uses natural metabolites as bait to reveal hidden allosteric pockets on disease-driving proteins that conventional drug discovery cannot reach. The company is advancing an oral AKT1-selective inhibitor for hereditary hemorrhagic telangiectasia (HHT) and a JAK2 V617F mutant-selective program for myeloproliferative neoplasms (MPNs).
Ensem Therapeutics
Ensem Therapeutics is a Waltham, Massachusetts clinical-stage biotech using its Kinetic Ensemble platform - a blend of AI/ML, molecular simulation, and experimental biophysics - to find cryptic and allosteric pockets in proteins that are otherwise considered difficult to drug. The company designs small-molecule precision medicines for cancer, with two lead programs: ETX-636, an allosteric pan-mutant-selective PI3Kalpha inhibitor and degrader for breast cancer (FDA Fast Track), and ETX-197/BG-68501, a selective CDK2 inhibitor partnered with BeiGene.
Indi Molecular
Indi Molecular is a Culver City, California biotech developing a synthetic alternative to antibodies called Protein Catalyzed Capture (PCC) agents - small, stable cyclic peptides built with click chemistry that bind protein targets with antibody-like specificity while behaving like small-molecule drugs. Spun out of Caltech chemist James Heath's lab in 2013, the company applies its platform to targeted oncology therapeutics and PET imaging agents, aiming to reach targets long considered undruggable, including KRAS G12D pancreatic cancer and ovarian cancer.
Flagship Pioneering
Flagship Pioneering is a Cambridge, Massachusetts life-sciences enterprise that invents, builds, and funds companies rather than merely investing in them. Founded in 1999 by Noubar Afeyan, the firm runs a systematic 'venture creation' engine - generating scientific hypotheses in-house, prototyping them in its own labs, and spinning validated concepts into independent operating companies in human health and sustainability. Its portfolio has produced more than 100 ventures, including Moderna, and it directs roughly $14 billion in capital.
Lyvgen Biopharma
Lyvgen Biopharma is a Shanghai-based clinical-stage immuno-oncology company founded in 2016 by veteran oncology scientist Dr. Jieyi Wang. It builds agonist antibodies - drugs that switch the immune system on rather than release its brakes - using a proprietary xLinkAb platform designed to concentrate immune activation inside tumors while limiting toxicity elsewhere. Its lead program, the CD137/4-1BB agonist antibody LVGN6051 (exlinkibart), is in Phase Ib/II trials in the US and China, including combinations with Merck's Keytruda, alongside a CD40 agonist (LVGN7409), a PD-1 blocker (LVGN3616), and bispecific candidates. Backed by IDG Capital and others through a Series C, Lyvgen runs R&D in Shanghai, a CMC/manufacturing center in Suzhou, and clinical and business development teams spanning China and the US.
Nan Ji
Nan Ji is co-founder, president and CEO of PAQ Therapeutics, a Burlington, Massachusetts clinical-stage biotech pioneering autophagosome-tethering compounds (ATTECs) to degrade KRAS and other 'undruggable' disease drivers. A Harvard-trained organic chemist with 30+ patent applications, he spent seven years at Novartis, helped build Kymera Therapeutics from seed stage as VP of Chemistry, and launched PAQ in 2020 to hijack the body's natural cellular clean-up machinery for cancer and neurodegenerative disease.
Sean X. Hu
Sean X. Hu is the President and CEO of Rapafusyn Pharmaceuticals, a Baltimore-based biotech spun out of Johns Hopkins that is building a library of natural-product-like macrocycles it calls RapaGlues. He is a genomics PhD turned Wharton MBA turned management consultant turned operator, with prior stops at Bristol Myers Squibb, CuraGen, Illumina, AT Kearney, IMS, Bionest, BioStrat, GlobalData, and Avotres. In September 2025 he closed an oversubscribed $44M Series A led by BioTrack Capital and Yonjin Capital to push RapaGlues into difficult-to-drug targets.
Fortitude Biomedicines
Fortitude Biomedicines is a Waltham, Massachusetts biotech that launched in early 2026 with $13M in seed financing to develop next-generation antibody-based therapies for autoimmune diseases and cancer. Its proprietary GLUE-DAC platform bolts molecular glue degraders onto antibody-drug conjugates, aiming to use catalytic targeted protein degradation to widen the therapeutic window of ADCs and overcome the resistance that has dogged the field. Its lead program, FORT-202, is a first-in-class T-cell targeting bispecific antibody for axial spondyloarthritis expected to reach the clinic in the first half of 2027.
Talus Bio
Talus Bio is a Seattle drug-discovery company going after transcription factors and other regulatory proteins long written off as 'undruggable.' Its MARMOT platform measures the human regulome directly in living cells using functional proteomics and high-throughput mass spectrometry, then feeds tens of millions of compound-target interactions into a lab-in-the-loop AI system to design small molecules that switch disease-driving genes on or off. Founded in 2020 by Alex Federation and Lindsay Pino, the company runs preclinical programs in chordoma, non-small cell lung cancer, and prostate cancer.
Joel Barrish
Joel Barrish is the President and CEO of Avilar Therapeutics, a Waltham, Massachusetts-based biotechnology company pioneering extracellular protein degradation. A medicinal chemist by training with a Ph.D. from Columbia University, Barrish spent 28 years at Bristol Myers Squibb rising to VP and Global Head of Discovery Chemistry, co-inventing blockbuster drugs including SPRYCEL (dasatinib) for leukemia. After stints as CSO at Achillion Pharmaceuticals and co-founder/President at Jnana Therapeutics, he founded Avilar in 2019 to build the ATAC and MTAC platforms - bifunctional molecules that hijack the body's own cellular recycling machinery to degrade disease-causing proteins outside the cell, a class of targets previously undruggable. Avilar has raised $75 million in total seed financing.
Abalone Bio
Abalone Bio is an Emeryville-based preclinical biotech building target-activating antibodies for diseases other drugs can't reach. Its FAST platform pairs engineered yeast cells with AI/ML to screen ~100 million antibodies per experiment for functional activity, not just binding, with an initial focus on G-protein coupled receptors (GPCRs) and metabolic disease.
Gordian Biotechnology
Gordian Biotechnology is a South San Francisco in vivo drug discovery company building a high-throughput screening platform that tests hundreds of gene therapies simultaneously inside animals that naturally develop age-related diseases. By pairing 'patient avatar' models with single-cell transcriptomics and machine learning (a stack the company calls Mosaic Screening + Pythia), Gordian aims to predict human clinical outcomes earlier and cure complex diseases of aging.
Kimia Therapeutics
Kimia Therapeutics is a Berkeley-based biotech building ATLAS, a drug-discovery platform that pairs nanoliter-scale high-throughput precision chemistry with machine learning and genome editing to map the druggable space at single-atom resolution. Spun out of Carmot Therapeutics in 2023 and backed by $55M in Series A funding, Kimia is using its chemical atlas to advance therapeutic programs in cancer and immunology.
Lycia Therapeutics
Lycia Therapeutics is a South San Francisco biotech building lysosomal targeting chimeras (LYTACs) - bifunctional molecules that drag disease-driving extracellular and membrane proteins into the cell's lysosomes for destruction. Founded in 2019 out of the lab of Nobel laureate Carolyn Bertozzi, Lycia is advancing a pipeline of autoimmune and inflammatory disease therapies, backed by $226M+ in venture capital and a strategic discovery collaboration with Eli Lilly.
Nextnet
Nextnet is a San Francisco-based life sciences AI platform that unifies the world's biomedical data into a semantic web, helping researchers find evidence-backed answers and connections across literature, genes, drugs, targets, and diseases. Its products - Nextnet Copilot and Nextnet Explorer - serve researchers at institutions including Harvard, MIT, UCSF, MD Anderson, and Columbia.
Plexium
Plexium is a San Diego precision medicine company designing small-molecule protein degraders - monovalent direct degraders and molecular glues - that switch off disease-driving proteins traditionally considered undruggable. Its DELTA Discovery platform runs ultra-high-throughput cell-based screening to find and optimize new degrader chemistry across cancer and neurological disease.
Prosetta Biosciences Inc
Prosetta Biosciences is a San Francisco biotech using a proprietary cell-free protein synthesis and assembly (CFPSA) platform to discover small-molecule 'assembly modulators' - drugs that disrupt the multi-protein complexes behind viral infection, cancer, and neurodegeneration. Founded by UCSF physician-scientist Vishwanath Lingappa, the company is advancing candidates against ALS/FTD, Alzheimer's, pan-cancer targets, and antivirals.
Switch Therapeutics
Switch Therapeutics is a South San Francisco preclinical biotech building conditionally activated siRNA (CASi) therapies that only switch on inside the cell types where they're needed. The platform - born from research at Caltech, Harvard and City of Hope - aims to make RNA interference safe enough for the brain, with a lead program targeting APOE for Alzheimer's disease.
Atomic AI
Atomic AI is a South San Francisco biotechnology company fusing machine learning with structural biology to unlock RNA drug discovery. Its platform pairs in-house wet-lab chemical-mapping data with deep learning models - including ATOM-1, a foundation model for RNA structure, and PARSE, its RNA structure exploration platform - to find structured, ligandable RNA motifs and design selective small molecules and RNA-based medicines for targets long considered undruggable.
Autobahn Therapeutics, Inc.
Autobahn Therapeutics is a San Diego clinical-stage biotech building small-molecule medicines that reach the brain on purpose. Using a brain-targeting chemistry and prodrug platform, the company tunes where a drug goes - central versus peripheral - to unlock validated CNS biology that has been hard to drug safely. Its lead program, elunetirom (ABX-002), is an oral, once-daily, brain-penetrant CNS thyroid hormone receptor agonist in Phase 2 trials for major depressive disorder and bipolar depression, backed by more than $200 million in venture funding and a 2026 FDA Fast Track designation.
Avelos Therapeutics
Avelos Therapeutics is a Seoul-based clinical-stage oncology biotech founded in 2021 that designs small-molecule drugs for 'undruggable' cancer targets using synthetic lethality, DNA damage response (DDR), and cell-cycle biology. Its lead program, AD1208, is a first-in-class oral MASTL kinase inhibitor now in a Phase 1/2a solid-tumor trial. Backed by KRW 30 billion (~$21.7M) across seed, Series A, and a 2024 Series B, the roughly nine-person team is building a biomarker-driven pipeline of four anti-cancer candidates aimed at a future KOSDAQ listing.
Cajal Therapeutics
Cajal Therapeutics (originally launched as Cajal Neuroscience) is a Seattle biotechnology company developing novel medicines to restore biological homeostasis, with a focus on neurodegenerative diseases, anemias of inflammation, and iron-related disorders. Built on a platform that pairs integrative human genetics, high-throughput functional genomics, and industrialized whole-brain imaging with deep neuroscience expertise, the company aims to systematically validate disease targets at unprecedented scale and translate them into small molecule and RNA therapeutics. It launched in November 2022 with a $96 million Series A and counts a roster of celebrated neuroscientists among its co-founders.
Concerto Biosciences
Concerto Biosciences is a Cambridge, Massachusetts microbiome company that turns microbial ecology into health products. Its patented kChip platform screens millions of microbial interactions in nanoliter droplets to discover 'ensembles' - small, defined groups of microbes that work together to shepherd a damaged microbial community back to health. The company's lead program, ENS-002, is a topical live biotherapeutic for atopic dermatitis that suppresses Staphylococcus aureus without nuking the skin's helpful microbes. Founded in 2020 by scientists out of MIT and the Broad Institute, Concerto pairs ultra-high-throughput biology with AI to make microbial communities predictable and engineerable.
Delphia Therapeutics
Delphia Therapeutics is a Cambridge, Massachusetts biotech pioneering a new field of cancer biology it calls activation lethality - the idea that cancer cells with oncogenic mutations live dangerously close to the upper limit of how much pathway activity they can tolerate, and can be pushed past it. Rather than blocking cancer signals like most drugs, Delphia designs targeted activators that overload cancer's stress pathways until the cell dies, aiming for durable benefit and a way around drug resistance. Founded in 2024 by Kevin Marks, Bill Sellers and Mike Dillon, it launched with a $67 million Series A led by GV, Nextech Invest, Polaris Innovation Fund and Alexandria Venture Investments.
Epirium Bio
Epirium Bio is a San Diego-based clinical-stage biopharmaceutical company developing orally available small molecules that target PGE2 signaling to restore tissue homeostasis, reduce inflammation, and stimulate regeneration. Its lead candidate, MF-300, is a first-in-class oral 15-PGDH enzyme inhibitor advancing toward a Phase 2b trial for sarcopenia, the age-related loss of muscle strength for which no FDA-approved therapy currently exists.
Gate Bioscience
Gate Bioscience is a Brisbane, California biotech building a brand-new class of medicines it calls Molecular Gates: oral small molecules that stop disease-causing proteins from ever leaving the cell. Instead of chasing proteins after they are secreted into the body, Gate's drugs bind Sec61, the single channel every one of the roughly 4,000 human secreted and membrane proteins must pass through, and selectively block a target protein so the cell degrades it. Founded in 2021 and emerged from stealth in 2023 with $60M, the company has raised about $135M total and is pushing its lead programs toward IND-enabling studies and Phase 1 trials.
Immetas Therapeutics
Immetas Therapeutics is a New Jersey biotech building drugs that target the chronic, low-grade inflammation that drives aging and age-related disease - what scientists call 'inflammaging.' Founded in 2018 by drug-development veteran J. Gene Wang and Harvard aging researcher David Sinclair, the company designs bispecific antibodies and other biologics to reprogram the innate immune system, aiming to treat age-related cancers and inflammatory and autoimmune disease. It raised an $11M Series A from Morningside Ventures in 2020 and partners with GC Biopharma on mRNA therapeutics.
Nilo Therapeutics
Nilo Therapeutics is a New York-based biotechnology company developing a new class of medicines that target the brain-body neural circuits regulating the immune system. Building on discoveries that identified specific vagal neurons controlling systemic inflammation, Nilo aims to restore immune homeostasis centrally rather than broadly suppress the immune system, offering a differentiated approach to treating autoimmune and inflammatory disease. The company launched out of stealth in October 2025 with a $101 million Series A.
Prellis Biologics
Prellis Biologics is a Berkeley, California biotech that 3D-prints human lymph node organoids to grow real human antibodies in a dish. Its EXIS platform uses holographic two-photon laser bioprinting plus AI to recreate the human immune response in vitro, discovering fully human antibody therapeutics faster and cheaper than animal-based methods, including against difficult targets like GPCRs where mouse models fail.
Treeline Biosciences
Treeline Biosciences is a Watertown, Massachusetts biotech founded in 2021 by Loxo Oncology founder Josh Bilenker and former Novartis oncology head Jeff Engelman. It pairs in-house wet-lab R&D with leading-edge computational tools to invent cancer medicines reliably and repeatedly, choosing targets by what the technology can reach rather than by therapeutic fashion. After operating largely in stealth, Treeline has raised more than $1.1 billion and moved three programs - a BCL6 degrader, a pan-KRAS inhibitor, and an EZH2 inhibitor - into Phase 1 trials. In June 2026 it agreed to go public through an all-stock reverse merger with Standard BioTools, with the combined company set to trade on Nasdaq as 'TRLN'.