Led · Grant · Jun 2026

Scribe Therapeutics engineers CRISPR-based technologies (including its X-Editor, XE) to create durable, potentially one-time genetic medicines aimed at key lipid drivers of atherosclerotic cardiovascular disease. Its lead candidate, STX-1150, is a liver-targeted therapy designed to epigenetically silence PCSK9 to reduce LDL-C without permanent DNA changes. The company’s cardiometabolic preclinical pipeline includes STX-1200 to lower lipoprotein(a) and STX-1400 to target APOC3 and durably lower triglycerides. Scribe’s XE platform is described as compact and highly engineered for enhanced activity, specificity, and deliverability across multiple editing modalities. The company is headquartered in Alameda, California, and has formed strategic collaborations with Sanofi and Eli Lilly. Scribe was co-founded by Jennifer Doudna and is advancing its programs toward clinical development with the goal of preventive, durable genetic medicines.

Led · Grant · Apr 2025

Ray Therapeutics is a Berkeley, California–based clinical-stage biopharmaceutical company developing optogenetic gene therapies to restore vision in patients with severe retinal degenerations. Its lead program, RTx-015, is an optogenetic gene therapy delivered as a single intravitreal injection and is being evaluated in patients with retinitis pigmentosa. The company’s second clinical-stage program, RTx-021, targets retinal bipolar cells and is designed for macular diseases such as Stargardt disease and geographic atrophy (GA AMD). RayTx’s approach delivers a bioengineered, highly light-sensitive protein to targeted retinal cells with the aim of improving visual function regardless of underlying genetic mutation. The company recently received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for RTx-015, and it plans to use recent financing to advance late-stage clinical development and commercial readiness.

Led · Grant · Feb 2025

Entos Pharmaceuticals develops genetic medicines using its proprietary, non-viral Fusogenix PLV delivery platform. The Fusogenix PLV platform is formulated with FAST proteins to enable direct fusion delivery of RNA, DNA and gene‑editing therapies and is described as combining the best aspects of viral and non‑viral approaches. Entos says the platform is suitable for a wide range of genetic medicines and is committed to advancing next‑generation therapies to improve patients' lives. The company will use CureDuchenne Ventures' initial $1M investment to create a muscle‑targeting therapeutic to deliver full‑length dystrophin for Duchenne muscular dystrophy (DMD). Entos plans to share information about the platform and its Duchenne program at the CureDuchenne 2025 FUTURES National Conference. Entos was founded in 2016 and is headquartered in Edmonton, Canada, with wholly owned U.S. and U.K. subsidiaries in San Diego and London. Entos Pharmaceuticals develops next-generation nucleic acid therapeutics using its proprietary Fusogenix PLV proteolipid vehicle platform. The Fusogenix PLV system uses FAST proteins to enable direct fusion delivery of nucleic acids to target cells. Entos plans to build a 103,000-square-foot R&D centre and a GMP biomanufacturing facility in Edmonton to develop, scale up, produce, and commercialize Fusogenix PLV therapeutics. The $198.5 million project is supported by $77.5 million in government funding. Entos has partnerships with Eli Lilly and collaborators including Oisin Biotechnologies, OncoSenX, and Aegis Life and aims to foster academic and industry collaborations in Canada. The company was founded in 2016 and is headquartered in Edmonton, with U.S. and U.K. subsidiaries in San Diego and London. Entos Pharmaceuticals is a clinical-stage genetic medicines company that uses its proprietary Fusogenix PLV platform to deliver RNA, DNA, and gene‑editing therapies. Its lead program, ENTLEP001, is a systemically administered investigational gene therapy designed to express native human leptin for the treatment of congenital generalized lipodystrophy (CGL); the candidate is intended to be durable and redosable. Preclinical studies in a leptin‑deficient mouse model produced clinically relevant leptin expression, normalization of glucose and insulin levels, and reduced weight gain and food intake. The company says the Fusogenix PLV platform combines aspects of viral and non‑viral approaches and published animal safety, efficacy, and biodistribution data in Cell in September 2024. Entos has stated plans to advance a second product into the clinic and has partnered with global companies including Eli Lilly. The company was founded in 2016 and is headquartered in Edmonton, Canada, with U.S. and U.K. subsidiaries in San Diego and London.

Led · Grant · Sep 2024

Neurona Therapeutics is developing allogeneic, off-the-shelf regenerative neural cell therapy products intended to provide long-term targeted repair of the nervous system following a single administration. Its lead candidate, NRTX-1001, comprises GABAergic interneurons and is being evaluated in two ongoing open-label multicenter Phase 1/2 trials for drug-resistant unilateral and bilateral mesial temporal lobe epilepsy (NCT05135091 and NCT06422923). The company presented updated Phase 1/2 data showing in a five-subject lower-dose cohort a median seizure reduction of 75% at six months, with four of five subjects reporting >50% reduction and two subjects reporting durable >95% reduction at 16 and 21 months; NRTX-1001 has been well tolerated and neuropsychological testing suggests absence of neurocognitive impairment. The FDA granted NRTX-1001 Regenerative Medicine Advanced Therapy (RMAT) designation in June 2024 based on these positive clinical data. Neurona plans to expand indications to neocortical focal epilepsy and other CNS disorders. Financially, the company has received multiple grants from the California Institute for Regenerative Medicine (CIRM), with cumulative CIRM commitment of $18.5M across five grants, and completed a $120M private financing in February 2024 co-led by Viking Global Investors and Cormorant Asset Management. Neurona Therapeutics is a San Francisco–based clinical-stage biotherapeutics company focused on developing regenerative, allogeneic, off-the-shelf cell therapy candidates with single-dose curative potential. Its candidates are designed to provide long-term repair of dysfunctional neural networks across multiple neurological indications. The company's lead investigational candidate, NRTX-1001, is being evaluated in an ongoing open-label, single-arm Phase I/II clinical trial for treatment of drug-resistant mesial temporal lobe epilepsy (MTLE). NRTX-1001 also has potential application in Alzheimer’s disease and other disorders of the nervous system. Led by CEO Cory R. Nicholas, Ph.D., Neurona intends to use newly raised capital to advance its wholly-owned pipeline of off-the-shelf cell therapies. The company recently raised $120M and announced that Raymond Kelleher, M.D., Ph.D., of Cormorant will be joining its board of directors. Neurona Therapeutics develops neural cell therapies intended to provide long-term repair of dysfunctional neural networks in chronic neurological disorders. Its lead candidate, NRTX-1001, is a neuronal cell therapeutic derived from human pluripotent stem cells comprising GABA-secreting interneurons designed as a one-time administration to provide local long-term inhibition. The company is advancing wholly-owned, off-the-shelf neuronal, glial, and gene-edited cell therapy candidates across multiple indications, initially targeting temporal lobe (focal) epilepsy. Neurona plans a Phase 1/2a clinical study for NRTX-1001 planned to launch later this year. The company completed a $41.5M financing that will be used to advance the pipeline; total proceeds raised to date are $135 million. Neurona describes its platform as based on a novel neural cell lineage with single-dose curative potential. Neurona Therapeutics is a pre-clinical biotechnology company focused on the transplantation of selected neurons to treat intractable neurological diseases. The company develops regenerative cell-based therapeutics composed of unique cell compositions that can be precisely targeted and integrated into damaged neural circuits. Its approach emphasizes activity-dependent regulation once transplanted into host circuitry. Neurona was founded by neuroscientists and stem cell researchers from the University of California, San Francisco, including Arturo Alvarez-Buylla, Arnold Kriegstein, John Rubenstein and Cory Nicholas. The company is led by interim CEO Tim Kutzkey and launched with a $23.5M Series A financing. Neurona is based in South San Francisco, CA and remains in the pre-clinical stage.

Led · Grant · Jul 2024

Senti Biosciences develops next‑generation cell and gene therapies using a proprietary Gene Circuit platform to create therapies with enhanced precision and control. Its wholly owned pipeline utilizes off‑the‑shelf chimeric antigen receptor natural killer (CAR‑NK) cells outfitted with Gene Circuits to target challenging liquid and solid tumors. SENTI‑202 is a potential first‑in‑class Logic Gated off‑the‑shelf CAR‑NK product candidate designed to selectively target CD33 and/or FLT3 expressing hematologic malignancies while sparing healthy bone marrow cells, combining OR and NOT gates plus calibrated‑release IL‑15 technology. The Phase 1 dose‑finding trial of SENTI‑202 is ongoing, enrolling adult patients with relapsed/refractory CD33 and/or FLT3 expressing hematologic malignancies at sites in the United States and Australia. Initial efficacy data from the trial is anticipated by year‑end 2024, with initial durability data expected in 2025. Financially, Senti Bio announced an $8 million grant from the California Institute for Regenerative Medicines to support SENTI‑202 clinical development; the company is publicly listed (Nasdaq: SNTI) and has partnerships with Spark Therapeutics and BlueRock Therapeutics. Senti Bio engineers proprietary gene circuits to program cell and gene therapies, using off-the-shelf chimeric antigen receptor natural killer (CAR-NK) cells outfitted with these circuits. Its lead programs include SENTI-202, an OR+NOT logic-gated CAR-NK candidate for acute myeloid leukemia, and SENTI-301, a multi-armed CAR-NK candidate for hepatocellular carcinoma. The company plans to file Investigational New Drug (IND) applications for both candidates in 2023 and has presented preclinical proof-of-concept data at ASGCT, AACR and ASH. Senti Bio has executed partnerships with Spark Therapeutics and BlueRock Therapeutics to advance its platform beyond oncology. The company is based in South San Francisco, Calif., and emphasizes programmable biological logic to improve therapeutic precision, durability and safety. Senti Biosciences uses synthetic biology to engineer gene circuits that program cells to sense their environment, perform logic, and produce therapeutic functions for improved cell- and gene-based medicines. Its proprietary platform includes logic gates, small-molecule regulators, combinatorial payloads and synthetic promoters, and the team has designed, built and tested thousands of gene circuits. Senti Bio is building an internal pipeline focused on next-generation allogeneic CAR-NK cell therapies, with lead candidates SENTI-202 for acute myeloid leukemia and SENTI-301 for hepatocellular carcinoma, plus additional undisclosed solid-tumor targets. The platform is intended to be deployable across multiple delivery modalities and therapeutic areas, including immunology, neuroscience, cardiovascular disease, regenerative medicine and genetic diseases. Proceeds from a recent financing will support preclinical oncology programs, IND-enabling studies (planned in 2021), expansion of the gene circuit platform into additional modalities, and scaling clinical manufacturing including process development and design of a cGMP-compliant facility for off-the-shelf CAR-NK products. The company communicated the financing in a release citing South San Francisco, Calif., and Leverkusen, Germany. Senti Biosciences develops programmable genetic circuits inserted into human-derived cells to create adaptive therapies that sense, respond to, and be controlled within localized disease environments. The company’s first therapeutic focus is cancer, where engineered cells are designed to locate tumors and selectively trigger an immune response after IV infusion. Senti bases its approach on decades of academic research and on gene‑editing technologies like CRISPR to write new “software” for cellular behavior. The team was assembled from leading labs (MIT, Wyss Institute, MD Anderson, Boston University, ETH Zurich, and others) during three years in stealth. Senti intends to develop therapies tailored to individual biology and to partner with other companies to address additional diseases. The company recently completed a major financing to support these efforts.