The 9 hottest private biotech companies in San Diego

With its enviable blend of sunny weather and beautiful beaches, San Diego stands out as one of the biggest hubs in the U.S. for biotech companies, boasting a vibrant ecosystem that nurtures innovation. In this article, we spotlight nine of the city’s leading players in the biotech sector.

The biotech scene in San Diego began to blossom between 1955 and 1965 when leading research institutions such as the Salk Institute, Scripps, and the University of California San Diego (UCSD) were founded close to one another. Along with these organizations, there are now also many giants of the biotech and pharma industry with a presence in the area, such as Pfizer, Illumina, and Thermo Fisher Scientific.

San Diego has grown into a huge center for biotech companies largely thanks to relatively affordable lab spaces, access to a strong network of biotech investors, and a skilled bio-economy workforce. It rivals other big biotech U.S. hubs, including South San Francisco, Los Angeles, Research Triangle Park in North Carolina, Florida, Chicago, and Boston. 

There is a host of biotech companies in San Diego working on world-changing innovations – with many based in the neighborhood of La Jolla – fueled by investments from life sciences venture capital (VC) firms, including local players Boxer Capital and Takeda Ventures.

Artiva Biotherapeutics

Artiva Biotherapeutics Inc. specializes in the development of cell therapies for cancer that are based on a type of tumor-hunting immune cell called natural killer (NK) cells. While approved CAR-T cell therapies for treating cancer derive their cells from the patient – which is a long and costly process – Artiva develops its NK cell therapies from the cells of donated umbilical cord blood, meaning that the manufacturing process is cheaper and the therapy can be delivered quickly to the patient.

Artiva’s lead candidate, AlloNK, is an allogenic, non-genetically modified NK cell therapy designed to enhance the effect of monoclonal antibodies or NK cell engagers. In the company’s lead project, AlloNK is being evaluated in a phase 1/2 clinical trial, which is designed to assess the safety and clinical activity of the therapy, both alone and in combination with the anti-CD20 monoclonal antibody, rituximab, in patients with relapsed or refractory B-cell non-Hodgkin lymphoma. 

The San Diego biotech company also recently received U.S. Food and Drug Administration (FDA) fast track designation for AlloNK for the treatment of the autoimmune condition, lupus nephritis. The therapy is being investigated in a trial to assess the safety and clinical activity of AlloNK in combination with anti-CD20 antibodies in patients with lupus nephritis who have relapsed or did not respond to previous standard-of-care treatment approaches. 

Artiva’s pipeline also includes CAR-NK candidates targeting both solid and hematopoietic cancers. 

Aspen Neuroscience

Aspen Neuroscience develops cell replacement therapies that are personalized to individual patients with neurological diseases. The cell therapies are derived from patients’ skin cells, which are then reprogrammed in the lab to become induced pluripotent stem cells that can mature into new neurons. 

Aspen bagged $147.5 million in a series B round in May 2022, allowing it to advance the development of its lead cell therapy, ANPD001, which is designed to tackle Parkinson’s disease by replacing nerve cells that are killed off by the disease. While there are multiple stem cell therapies in development for treating Parkinson’s, many are sourced from healthy donors, which could lead to the immune system rejecting the graft. Aspen’s approach of sourcing the cells from the patient could avoid this immune rejection and make it easier for the graft to succeed.

In October 2023, Aspen announced that the FDA had granted fast track designation for ANPD001. The San Diego-based biotech company is now planning to initiate a first-in-patient phase 1/2a clinical trial of the therapy for participants with moderate to severe Parkinson’s. 

The other candidate in development at Aspen – known as ANPD002 – is a neuron replacement therapy for Parkinson’s that has been genetically altered so that the cells don’t contain risk factor genes for the disease.

Belharra Therapeutics

After debuting in January 2023 with $130 million in funding, drug discovery company Belharra Therapeutics is trying a new approach to target and ligand identification. According to the company, only 3% of the proteome is targeted with small molecule therapeutics, leaving a big catalog of targets on drug developers’ wish lists and a vast proportion of the proteome unexplored. But, using its platform, Belharra wants to identify functional and actionable non-covalent, small molecule drug candidates for any binding site, on any protein, in any conformational state, in any cell type. 

The company’s integrated chemoproteomic-based drug discovery engine addresses the limitations of traditional screening approaches through a proprietary library of photoaffinity-based chemical probes (tools that allow the capture of protein targets in live cells) that explore protein-ligand interactions in the native environment of the cell. The platform uses photoaffinity-based labeling (a method for covalently binding chemical tags to the active sites of protein molecules) to “trap” these unique non-covalent protein-ligand interactions. This ultimately creates the potential to identify next-generation therapeutics for previously difficult-to-treat conditions. 

Announced in January last year, at the same time that the biotech company debuted, Belharra has a multi-year collaboration with Genentech that employs the San Diego company’s platform to discover and develop small molecule therapies in multiple therapeutic areas such as oncology, immuno-oncology, autoimmune, and neurodegenerative diseases.

Capstan Therapeutics

Listed on Labiotech’s ‘companies to watch in 2024’, Capstan Therapeutics launched in 2022 with $165 million to develop targeted in vivo RNA-based therapies. Its core platform technology comprises targeted lipid nanoparticles (tLNPs) that are composed of LNPs conjugated with a recombinant protein binder, such as a monoclonal antibody. tLNPs are designed to deliver payloads, including mRNA or gene editing tools, capable of reprogramming specific cell types in vivo. This method could offer a more efficient and potentially less costly alternative to current ex vivo CAR-T cell therapies.

Capstan’s technology has the potential to generate transformative therapies with possible applications across a broad range of disease areas, including autoimmune disorders, oncology, fibrosis, and monogenic blood disorders. 

The company announced in October 2023, that it would be presenting preclinical data at the Society for Immunotherapy of Cancer (SITC) showing rapid and robust anti-tumor and anti-primary B cell activity in humanized mice by tLNP administration delivering an mRNA that encodes an anti-CD19 CAR. These data highlight the potential of Capstan’s technology. 

Fate Therapeutics

Fate Therapeutics wants to bring a pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune disorders. It has a platform that allows for iPSC cells to be genetically engineered and expanded in order to create off-the-shelf cell therapy drugs. The company’s cells of interest are the cells of the immune system and its cell therapy product candidate pipeline is comprised of natural killer (NK) cell and T cell product candidates. 

The San Diego-based biotech company has four clinical candidates designed to target cancer cells and one clinical candidate aimed at tackling autoimmune diseases. Its lead candidate is an NK cell therapy called FT576 intended for the treatment of multiple myeloma. The therapy targets the B-cell maturation antigen (BCMA) protein with the help of its proprietary CAR. It also contains a modified non-cleavable CD16 Fc receptor to improve antibody-dependent cell cytotoxicity and an IL-15 receptor fusion to enhance NK cell activity. 

The company’s most recent development, announced in January, was the initiation of a phase 1 trial for its fourth oncology candidate, known as FT825, in patients with HER2-expressing advanced solid tumors. 

Neurocrine Biosciences

Neuroscience company Neurocrine Biosciences has a broad pipeline of clinical candidates across neurology, neuroendocrinology, and neuropsychiatry indications. It also already has FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington’s disease, Parkinson’s disease, endometriosis, and uterine fibroids. 

One of the company’s lead candidates currently in development is called crinecerfont, which is intended for the treatment of congenital adrenal hyperplasia (CAH), a group of genetic conditions that result in an enzyme deficiency that alters the production of essential adrenal hormones. Crinecerfont is a selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed to reduce and control excess adrenal androgens through a steroid-independent mechanism. 

In December 2023, the therapy received breakthrough therapy designation from the FDA. This came after separate announcements from the company in September 2023 and October 2023 showed that phase 3 studies of crinecerfont had produced positive topline results in adults with CAH and children and adolescents with CAH, respectively. 

Poseida Therapeutics 

Based in San Diego, biotech company Poseida Therapeutics is advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases. Its approach to cell and gene therapies is based on its genetic editing platforms, including its non-viral piggyBac DNA Delivery System that can stably integrate DNA into the genome, its Cas-CLOVER Site-Specific Gene Editing System, and nanoparticle and hybrid gene delivery technologies.

The company’s pipeline includes allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors, as well as in vivo gene therapy candidates. Its lead candidate is known as P-BCMA-ALLO1, which is a CAR-T candidate targeting BCMA for the treatment of relapsed/refractory multiple myeloma. Just this month, the company announced that the FDA had granted orphan drug designation for P-BCMA-ALLO1.

P-BCMA-ALLO1 is being developed in partnership with Roche after the two companies announced a strategic global collaboration in 2022, with a focus on allogeneic CAR-T cell therapies for hematological malignancies. Furthermore, Astellas Pharma recently announced a strategic investment to support the advancement of Poseida’s mission to redefine cancer cell therapy. 

RayzeBio

RayzeBio operates within the thriving field of radiopharmaceuticals, developing solid tumor treatments based on radioactive molecules like actinium-225. The radioactivity released by actinium-225 has a very short range of just a few cell widths. When RayzeBio’s tumor-seeking drugs bind to a tumor target, the attached actinium-225’s powerful, short-range radiation can smash nearby cancer cells while sparing healthy tissue further from the tumor.

RayzeBio’s lead candidate is called RYZ101. It targets somatostatin receptor 2 (SSTR2), which is a protein that is over-expressed in gastroenteropancreatic neuroendocrine tumors (GEP-NETs) and extensive stage small cell lung cancer (ES-SCLC). Patients with SSTR-positive GEP-NETs who have previously been treated with lutetium-177-based somatostatin therapies are being enrolled in a phase 3 trial of the drug.

RayzeBio raised $160 million in a series D round in 2022 before overshooting expectations in September last year with a $358 million initial public offering (IPO). It was then announced last month that the San Diego biotech company had officially become a part of Bristol Myers Squibb (BMS) after the pharma giant completed its acquisition. 

Travere Therapeutics

Rare disease company Travere Therapeutics achieved an important milestone last year when the FDA granted accelerated approval for its therapy for primary IgA (immunoglobulin A) nephropathy. IgA nephropathy is a rare disorder that affects the kidneys and happens when IgA settles there. It is usually treated using angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs), but, with its approval, Travere’s drug became the first non-immunosuppressive therapy to treat the disease. 

The therapy, called FILSPARI, works by blocking the endothelin-1 and angiotensin II receptors, two critical pathways in the progression of IgAN. It is intended for patients who are at risk of rapid disease progression that will likely result in end-stage renal disease. The accelerated approval for the drug was granted on the basis that it is shown to reduce proteinuria, which in turn reduces the risk of kidney failure. Travere has recently submitted a supplemental new drug application to the FDA seeking full approval of FILSPARI.

The San Diego biotech company also has three clinical candidates in its pipeline targeting rare kidney and metabolic diseases. The most recent update from its pipeline came in December 2023, when the company announced it had initiated a phase 3 trial of its candidate for classical homocystinuria (HCU), a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS).

San Diego life sciences industry: a strong economic impact

According to the 2023 Life Science Economic Impact Report from Biocom California, life sciences employment grew by 8% overall in the San Diego cluster in 2022. Its life science workforce consists of more than 77,000 direct employees and continues to be a driving force in the local economy. Life sciences jobs in San Diego County paid an average of $144,000 yearly and represented a regional economic impact of $57.4 billion. These statistics show that the region continues to solidify its position as one of the country’s most important life science research destinations.