Over the last decade we have advanced disruptive ventures developing disease-modifying cellular medicines, several of which are now in clinical development. Correcting an underlying deficit within a cell or tissue can halt degeneration and potentially reverse the course of disease. More recently we have branched out to correct other kinds of deficits.

Status: Clinical | Partner: Bayer

Opsis Therapeutics pioneered the manufacturing of off-the-shelf, iPSC-derived retinal cells to treat late-stage inherited blindness. Following a strategic R&D alliance with BlueRock Therapeutics (Bayer AG) valued at over $70 million in initial commitments, Opsis advanced its lead program (OpCT-001) into the clinic. OpCT-001 creates a new paradigm for retinal repair by replacing lost photoreceptors. It has received FDA Fast Track designation and is currently dosing patients in the CLARICO trial (NCT06789445).

Status: Clinical | Partner: TCG

Kenai Therapeutics Kenai Therapeutics is revolutionizing the treatment of Parkinson’s disease through regenerative medicine. Backed by an $82 million Series A financing co-led by The Column Group, Kenai is advancing an allogeneic dopamine progenitor therapy (RNDP-001). The company has dosed the first patient in the Phase 1 REPLACE™ trial (NCT07106021), and received FDA Fast Track designation. RNDP-001 is designed to re-innervate the brain and restore motor function.

Status: Clinical | Partner: Novartis

Papillon Therapeutics is developing multi-systemic genetic medicines using engineered hematopoietic stem cells (HSCs) to treat inherited diseases. The company’s platform modifies the body’s own immune cells to deliver therapeutic proteins to hard-to-reach organs like the brain and heart. Papillon’s Cystinosis program is partnered with Novartis (Phase 1/2), and its internal lead for Friedreich’s Ataxia (PPL-001) has received FDA Orphan Drug and Rare Pediatric Disease designations.

Status: Preclinical | Partner: BIVF

ProTgen is developing first-in-class medicines for immuno-oncology and immune health by re-activating the thymus. The company’s proprietary bi-functional Notch activators drive the differentiation of hematopoietic stem cells into progenitor T-cells (pro-T). This therapy aims to restore the immune repertoire in oncology patients undergoing bone marrow transplant and reverse the immunosenescence associated with aging.

Status: Preclinical | Partner: USC

StemCardia is developing therapies to reverse heart failure, the leading cause of death worldwide. In collaboration with the University of Southern California (USC), StemCardia lead candidate (SCM-101) is a potentially curative therapy composed of billions of iPSC-derived cardiomyocytes produced in scalable 3D bioreactors. These cells to re-muscularize the heart and restore pumping function in primate models.

Status: Preclinical | Partner: FFB RD Fund

Agnos Therapeutics is advancing a next-generation retinal replenishment therapy to treat intermediate stage inherited blindness. The company’s lead candidate (AGN-001) is designed to rescue dying photoreceptors by transferring healthy cytoplasm and organelles from donor cells. With the support of Foundation Fighting Blindness (FFB) Retinal Degeneration Fund, this mutation-agnostic approach offers the potential of a rapidly acting treatment for the millions of patients.

Status: Preclinical | Partner: Reboot

Vascugen is a company focused on advancing the pipeline of cell replacement therapies targeting microvascular diseases. Leveraging innovations in induced pluripotent cell (iPSC) generation and vascular cell differentiation, Vascugen is developing cell therapies for patients with severe ischemic conditions of the limb, lung, and retina.

Status: Preclinical | Partner: Reboot

Dianomi Therapeutics was a preclinical stage biopharmaceutical company advancing a pipeline of next generation treatments for inflammatory diseases, initially targeting osteoarthritis. The company’s proprietary Mineral Coated Microparticle (MCM) technology enables formulation of proven drugs for improved delivery, stability, and function.

Status: Commercial | Partner: St.Jude

RPRD Diagnostics (Right Patient Right Drug) is a precision medicine company offering clinical pharmacogenomics (PGx) testing and analysis services. The company specializes in comprehensive PGx and tailored panels, such as the CNT Panel for identification of leukemia patients at risk for toxicity from thiopurine drugs. These tests enable physicians to consider a patient’s unique genetic profile when recommending drug treatment.

Status: Seed | Partner: Nvidia Inception

CapyBio is building the "GPS" for cell engineering to supply pure, scalable human cells for drug discovery and safety testing. By combining proprietary AI/ML tools (CellOracle and Capybara) with high-throughput biology, CapyBio dramatically improves the manufacturing and maintenance complex cell types like adipocytes and hepatocytes, addressing the critical failure rate of animal models in drug development.

Status: Seed | Partner: Nvidia Inception

pr1or.art is an AI-powered strategic defense platform for the intellectual property (IP) landscape. By using Generative AI (AWS Bedrock) to create and publish technical disclosures ("prior art") at scale, the platform preemptively blocks competitors from filing broad, obstructive patents. It offers an asymmetric economic advantage, delivering Freedom to Operate (FTO) support for <$100 per asset.

Status: Seed | Partner: Microsoft AI Co-Innovation

ThoughtFence is the digital picket fence for the creator economy. Built on secure Azure PaaS infrastructure, it provides a verifiable, timestamped workspace for innovators. With integrated "Agentic Novelty" AI, ThoughtFence helps creators structure their ideas into defensible assets, ensuring that ownership is clear and indisputable from the moment of inception.

2025.12.15 - Kenai Therapeutics Announces First Patient Dosed in Phase 1 REPLACE™ Clinical Trial of Neuron Replacement Cell Therapy RNDP-001 for Idiopathic Parkinson’s Disease

2025.07.08 - BlueRock Therapeutics announces first patient receives investigational therapy in Phase 1/2a clinical trial of OpCT-001 for the treatment of primary photoreceptor diseases

2025.02.27 - BlueRock Therapeutics receives FDA Fast Track Designation for OpCT-001 for the treatment of primary photoreceptor diseases

2024.10.21 - Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-002 for the Treatment of Danon Disease

2024.10.15 - Papillon Therapeutics Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

2024.10.08 - USC launches collaboration with StemCardia to advance heart regeneration therapies

2024.10.01 - Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-002 for the Treatment of Danon Disease

2024.09.30 - StemCardia Licenses Technologies for Heart Regeneration Developed at UW School of Medicine

2024.09.17 - Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

2024.09.03 - BlueRock Therapeutics announces FDA clearance of IND application for investigational iPSC-derived cell therapy OpCT-001 for the treatment of primary photoreceptor diseases

2024.04.15 - NuFund Venture Group Invests in Papillon Therapeutics to Advance Multi-Systemic Genetic Medicines

2024.03.13 - BioCardia and StemCardia Announce Biotherapeutic Delivery Partnership

2024.01.23 - Kenai Therapeutics Announces $82 Million Series A Financing to Advance Next Generation Allogeneic Cell Therapies for Neurological Diseases

2024.01.23 - BlueRock Therapeutics exercises exclusive option to license iPSC cell therapy candidate OpCT-001 for treating Primary Photoreceptor Diseases from FUJIFILM Cellular Dynamics and Opsis Therapeutics

2023.02.14 - Ryne Biotechnology Awarded $4 Million from the California Institute for Regenerative Medicine

2022.08.31 - First U.S. patient receives autologous stem cell therapy to treat dry AMD

2021.05.17 - BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics Enter into a Strategic R&D Alliance to Discover and Develop Cell Therapies for Eye Diseases

2021.02.09 - US Capital Global Launches $7MM Preferred Stock Offering for Dianomi Therapeutics, Inc.

2020.02.11 - Dianomi Therapeutics Exclusively Licenses Nucleic Acid Technologies from the Wisconsin Alumni Research Foundation

2019.07.28 - RPRD Diagnostics and St. Jude Children’s Research Hospital Work Toward Racially Diverse Pharmacogenomics Testing to Meet Goal of Precision Medicines

2019.02.04 - Ligand Announces Investment in Dianomi Therapeutics

2018.11.16 - IU Philanthropic Venture Fund Invests in Regenerative Medicine Company Vascugen

2018.05.01 - Vascugen Licenses Stem Cell Technology for Blood Vessel Regeneration Developed at IU School of Medicine

2017.10.19 - RPRD Diagnostics Partners with St. Jude Children's Research Hospital to Offer Comprehensive Pharmacogenetics Testing

2017.02.13 - RPRD Diagnostics Announces Strategic Collaboration with Children's Minnesota to Advance Clinical Pharmacogenomics

2016.10.17 - RPRD Diagnostics Launches to Bring Comprehensive Pharmacogenetics Testing to Routine Clinical Practice

2016.9.28 - Cellular Dynamics International Launches Opsis Therapeutics to Develop Cell Therapies for Treatment of Retinal Diseases