Klotho Therapeutics has a patented protein, a myth-sized ambition and a very human bottleneck: animal evidence is intriguing, but patients need manufacturing, dosing and trials - not mythology.
The medicine was familiar. The delivery problem was not. Neurelis spent more than a decade making diazepam work as a practical nasal rescue treatment - then showed how a narrow patient problem can become a platform, a pipeline and a profitable commercial company.
80% of cancer compounds that reach clinical trials fail. Yatiri Bio thinks the problem is that drug developers have been reading the wrong molecules - and it built a proteomics platform to prove it.
One movement-disorder medicine turned a San Diego research shop into a multibillion-dollar biopharma. Now Neurocrine is spending that advantage on rare endocrine disease, psychiatry and the difficult biology between brain and body.
Floré, built by San Diego biotech Sun Genomics, makes probiotics the slow way: you mail in a stool sample, an accredited lab reads the whole genome of your gut microbiome, and an algorithm trained on tens of thousands of prior formulations builds a supplement matched to the bacteria you actually carry. Founded in 2016 by former Illumina and LabCorp genomicist Sunny Jain, the company sells a subscription that re-tests and re-formulates over time, and has published peer-reviewed research pairing its precision synbiotics with gut and autism-spectrum outcomes.
MBrace Therapeutics is a clinical-stage oncology company building antibody-drug conjugates (ADCs) against hard-to-drug cancer targets. Its proprietary SPARTA platform screens antibodies directly inside living tumors to surface antigens that are cancer-specific, accessible, and internalizing - the traits an ADC needs to deliver its toxic payload where it counts. Founded in 2020 and headquartered in San Diego, the company raised an $85M Series B in 2023 (led by TPG) to advance a pipeline led by MBRC-101, a first-in-class ADC targeting the EphA5 receptor now in Phase 1/2 clinical trials.
Aizen Therapeutics is a San Diego, AI-driven biotech spun out of Caltech that designs Mirror Peptides - synthetic drugs built entirely from D-amino acids, the mirror image of the L-amino acids that make up natural proteins. Its DaX platform pairs generative AI with structural biology to design these molecules from scratch, aiming for drugs with the potency of biologics but greater stability, lower immunogenicity, and the potential to be taken orally. The company emerged from stealth in November 2024 with a $13M seed round.
Alterome Therapeutics is a San Diego-based clinical-stage biopharmaceutical company building next-generation small molecule cancer drugs aimed at specific oncogenic mutations. Using its Kraken platform - a structure-guided, machine-learning approach to drug design - Alterome has advanced two precision oncology programs into the clinic: ALTA2618, an AKT1 E17K mutation-selective inhibitor, and ALTA3263, a KRAS-selective inhibitor covering roughly 90% of KRAS mutations including G12V and G12D.
858 Therapeutics is a clinical-stage biotechnology company in San Diego building small-molecule cancer drugs aimed at novel targets in DNA damage response, RNA modulation, and innate immunity. Its lead candidate, the oral PARG inhibitor ETX-19477, is in a first-in-human Phase 1/2 trial for advanced solid tumors and has won FDA Fast Track designation for BRCA-mutated, platinum-resistant ovarian cancer. Founded in 2019 by a serial team of San Diego drug developers, the company has raised $110 million across Series A and B financings.
BlossomHill Therapeutics is a San Diego clinical-stage biopharmaceutical company designing small-molecule precision medicines for cancer and autoimmune disease. Founded in 2020 by drug designer J. Jean Cui and biotech veteran Y. Peter Li - the team behind Turning Point Therapeutics (acquired by Bristol Myers Squibb in 2022) - the company is advancing a wholly-owned pipeline led by BH-30643, a first-in-class macrocyclic OMNI-EGFR inhibitor for EGFR-mutant non-small cell lung cancer, and BH-30236, a CLK inhibitor targeting aberrant RNA splicing in AML and high-risk MDS.
Light Horse Therapeutics is a San Diego biotech founded in 2023 that flips traditional drug discovery on its head. Using a proprietary 'function-first' platform that pairs systematic CRISPR-based gene editing with chemical biology, the company first pinpoints which exact regions of a disease-driving protein are functionally critical, then hunts for small molecules that hit those precise sites. Launched out of Versant Ventures' Inception Discovery Engine with a $62M Series A and a Novartis collaboration worth up to $1 billion in milestones, Light Horse is targeting some of oncology's toughest, historically 'undruggable' proteins.
Neomorph is a San Diego clinical-stage biotech building molecular glue degraders - small molecules that recruit the cell's own disposal machinery to destroy disease-driving proteins long written off as 'undruggable.' Founded in 2020 out of Deerfield Management with scientific founders who decoded how thalidomide-class drugs work, the company has assembled what it calls the world's largest proprietary molecular glue target space across a broad portfolio of E3 ubiquitin ligases. It has signed multibillion-dollar discovery pacts with AbbVie, Novo Nordisk, and Biogen, dosed its first patient with lead candidate NEO-811 in kidney cancer, and raised roughly $209M across Series A and B.
Pleno Inc. is a San Diego multi-omic instrument company rebuilding the economics of biological target detection. Its RAPTOR platform, powered by proprietary Hypercoding technology borrowed from telecommunications signal processing, can detect up to 10,000 targets in a single sample at PCR-like speed and cost. Founded by serial entrepreneur Pieter van Rooyen, the company is moving from R&D into commercial launch with backing from Deerfield Management and Foresite Capital.
Tentarix Biotherapeutics is a San Diego biotech building conditionally active, multispecific biologics that switch on only at the cells where they are needed. Its proprietary stack - the VH-Select stabilized human VH scaffold, Tentacles multifunctional molecules, and the FunctionSeq mammalian-display screening engine - lets the company discover therapies with high cell selectivity and a wider therapeutic index across oncology, immuno-oncology, and autoimmune disease. Backed by Versant Ventures, Samsara BioCapital, Amplitude Ventures, and Gilead Sciences, and partnered with AbbVie and Gilead.
Zafrens is a San Diego biotechnology company building an ultra-high-throughput single-cell platform that isolates, images, runs assays on, and sequences millions of individual cells per day. Its Z-Screen technology swaps the conventional 96-well plate for a credit-card-sized plastic chip holding 50,000 to 200,000 microwells, each with integrated imaging and multi-omic sequencing. By linking perturbation to genotype, phenotype and function at single-cell resolution, Zafrens compresses multiple stages of drug discovery into a single benchtop experiment - a 500x to 2,000x jump in the number of experiments a scientist can run at one timepoint.
Bloom Science is a San Diego clinical-stage biotech translating the biology of the ketogenic diet into oral live biotherapeutics that act through the gut-immune-brain axis. Built on microbiome research licensed from UCLA and powered by its IrisRx discovery platform, the company is developing BL-001, a once-daily bacterial therapy being studied in obesity and in drug-resistant seizures from Dravet syndrome, with a pipeline reaching toward ALS and neurodegenerative disease.
Mirador Therapeutics is a San Diego precision-medicine company building first- and best-in-class therapies for immune-mediated inflammatory and fibrotic diseases. Its Mirador360 engine fuses human genetics, multi-modal patient data, AI and advanced analytics to find novel targets, design combination therapies and identify the patients most likely to respond. Founded in 2024 by the former Prometheus Biosciences leadership team, Mirador launched with more than $400 million and has since raised over $650 million total.