Capstan Therapeutics Launches with $165 Million to Deliver on the Clinical Promise of Precise In Vivo Cell Engineering

Capstan | September 15, 2022 | Read time : 05:00 min

Capstan Therapeutics
Capstan Therapeutics, Inc., a biotechnology company dedicated to developing and delivering precise in vivo cell engineering to patients, launched today with $165 million in financing to build on the foundational insights of world-renowned leaders in mRNA and cell therapy, combining the power of cell therapy with the precision of genetic medicines to help bring safer, first-in-class medicines to more patients in multiple indications.

Capstan’s foundational precision in vivo engineering technology builds on research conducted in the laboratories of world-renowned mRNA and cell therapy scientists at the University of Pennsylvania.

Capstan’s funding includes a recently closed $102 million Series A financing led by Pfizer Ventures and joined by Leaps by Bayer, Eli Lilly and Company, Bristol Myers Squibb, Polaris Partners, Alexandria Venture Investments, and all existing investors, which follows a $63 million seed financing led by Novartis Venture Fund and OrbiMed and joined by RA Capital, and Vida Ventures in November 2021.

Advancing Breakthrough Therapies through Precision in vivo Engineering
The company plans to use the funds to further its mission of advancing the clinical promise of cell-based therapies by enabling precise in vivo engineering of cells with payloads necessary to benefit patients across multiple disease categories. Capstan’s modular platform leverages the biological and technological expertise of its founding scientists and includes proprietary targeted lipid nanoparticles (tLNP) technology, a suite of targeting moieties to mediate cell type-specific uptake, and disease-specific mRNA payloads aimed at directly engineering, or ablating pathogenic cells through in vivo generated CAR T cells.

Capstan is prioritizing programs based on the potential to transform clinical standards of care. The company’s initial efforts will focus on developing first-in-class in vivo CAR therapies, with the goal to deliver treatments in an outpatient setting, for patients who have diseases for which there are no effective treatments. Capstan also plans to leverage its precision delivery and engineering technology to advance new therapies for certain monogenic blood disorders.

Visionary Team of Accomplished Executives Poised to Deliver a Transformational Approach
Capstan is also pleased to announce that Laura Shawver, Ph.D., has joined the company as President and Chief Executive Officer. Dr. Shawver brings more than 20 years of experience in executive leadership positions in oncology and other serious diseases to this role, most recently serving as CEO of Silverback Therapeutics and President and CEO of Synthorx.

“Our ambition at Capstan is to invent new clinical paradigms through targeted in vivo reprogramming of cells,” said Dr. Shawver. “Our founding scientific and operational team is purpose-built to advance programs to the clinic that unite decades of combined experience in groundbreaking CAR therapies with the latest advances in mRNA delivery technology. We are also fortunate to have a distinguished investor syndicate that understands the cell therapy landscape and recognizes the potential of this innovative approach. I am thrilled to lead this team as we work toward making this vision a reality for patients.”

Laura is an exceptional leader with a well-established track record in drug development and passion for addressing unmet needs in oncology and other serious diseases. We are delighted to have someone of Laura’s caliber captain this exciting next chapter for Capstan to help unlock the potential of this transformative approach for patients.”

Michael Baran, Ph.D., Partner, Pfizer Ventures

Dr. Shawver joins an accomplished leadership team of industry veterans who have been at the forefront of cell and gene therapy, including Adrian Bot, M.D., Ph.D., Chief Scientific Officer, formerly Global Head and Vice President of Translational Medicine at Kite, a Gilead Company and previously Chief Scientific Officer at Kite Pharma; and Priya Karmali, Ph.D., Chief Technology Officer, who has over two decades of experience in the field of lipid nanoparticle mediated nucleic acid delivery technologies.

The company’s scientific founders include several of the experts from the University of Pennsylvania who authored two landmark studies establishing preclinical proof-of-concept for non-viral, in vivo CAR-T therapy that Capstan plans to develop and advance toward the clinic. A 2019 Nature publication demonstrated the preclinical use of ex vivo CAR-T cell therapy against FAP, a fibrosis-related target. A follow-on study published in Science earlier this year built on these earlier results and demonstrated the production of functional CAR-T cells in vivo in a mouse model following a single IV administration of an mRNA encoding an anti-FAP CAR packaged in CD5-targeted-LNPs. The scientific founders span diverse areas of research expertise

  • Preclinical translation – Jonathan Epstein, M.D. and Haig Aghajanian, Ph.D. (Dr. Aghajanian is now a Capstan employee.)
  • Cell engineering – Carl June, M.D. and Bruce Levine, Ph.D.
  • mRNA and targeted LNP technologies – Drew Weissman, M.D., Ph.D. and Hamideh Parhiz, Pharm.D., Ph.D.
  • Immunology and Fibrosis – Ellen Puré, Ph.D. and Steven Albelda, M.D.

“Capstan is uniting several recent life science technological advances in a manner that can hopefully unlock the potential of these technologies to develop new medicines for patients across a wider range of diseases,” said Drew Weissman, M.D., Ph.D., Co-Founder of Capstan, who serves as the Roberts Family Professor in Vaccine Research and Director of the Penn Institute for RNA Innovation in the Perelman School of Medicine at the University of Pennsylvania, and co-recipient of the 2021 Lasker-DeBakey Clinical Medical Research Award.

“Research conducted at Penn demonstrates the tremendous promise of harnessing mRNA and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo, potentially creating new treatment options,” said Jonathan Epstein, M.D., Chief Scientific Officer at Penn’s Perelman School of Medicine. “We believe this approach has the potential to make an important impact not only in oncology, but also in fibrosis and many other diseases. My fellow scientific co-founders and I all look forward to actively partnering with Capstan in our collaborative effort to develop medicines that may benefit patients around the world.”

About Capstan
Capstan Therapeutics is advancing precision in vivo cell engineering to develop therapeutics for a broad range of disease categories with unmet or underserved clinical need. The core technology comprises targeted Lipid Nanoparticles to enable engineering or ablation of pathogenic cells in the body. The company is combining the power of cell therapy with the precision of genetic medicines to develop new treatment options for patients for oncology, fibrosis, inflammation-related diseases, and monogenic blood disorders. The Company is co-founded by pioneers in the field of CAR-based immunotherapy, nucleic acid drug delivery, tLNPs, and regenerative medicine, including experienced industry leaders, as well as academic faculty members from the University of Pennsylvania. Capstan has a bicoastal presence with operations in San Diego, CA and Philadelphia, PA.


If scaling issues strike you as obvious or even boring, you should read “On Being the Right Size,” a 1926 essay by geneticist J.B.S. Haldane. It makes the case that a difference in scale—a seemingly simple matter—may demand subtle changes in form and function.


If scaling issues strike you as obvious or even boring, you should read “On Being the Right Size,” a 1926 essay by geneticist J.B.S. Haldane. It makes the case that a difference in scale—a seemingly simple matter—may demand subtle changes in form and function.

Related News


Synthego Introduces CRISPR Discovery Partners to Streamline CRISPR-Driven Drug Discovery Research

Synthego | March 17, 2023

Synthego Corp., a leading genome engineering solutions provider, announced today the launch of its service partner ecosystem, CRISPR Discovery Partners, designed to accelerate drug discovery by providing a seamless research workflow from CRISPR editing to phenotypic data and downstream services. The ecosystem brings together high-value partners including Arctoris, BrainXell, Curia, PhenoVista, and Pluristyx to offer a coordinated solution for CRISPR-driven drug discovery. The CRISPR Discovery Partner ecosystem creates an integrated workflow starting with Synthego's CRISPR Engineered Cells through functional readouts, providing researchers with a faster, more efficient, and more reliable way to move their discoveries forward. Researchers can access the full range of genome engineering services through Synthego, as well as downstream services including small molecule screening, cell-based assays, high content imaging, iPSC differentiation, and multi-omics through partners. The result is a streamlined drug discovery process that can help accelerate the development of life-saving treatments. Synthego's CRISPR Discovery Partners include Arctoris, a tech-enabled drug discovery service company, combines wet lab automation and machine learning with a world-class team of experienced drug discovery researchers to enable the accelerated development of small molecule, biologics, and cell therapy assets. BrainXell provides differentiation services to generate high-purity, iPSC-derived human neurons, astrocytes, and microglia for research use with a focus on drug discovery. BrainXell also offers fibroblast reprogramming and select downstream assay services. Curia, a global contract research, development and manufacturing organization (CDMO), offers products and services from discovery through manufacturing to help customers transform R&D results into real-world impact. PhenoVista, a San Diego-based CRO, specializes in modern, human, cell-based in vitro assay development and analysis. Internationally recognized for developing and implementing both fully bespoke projects and pre-validated Ready-2-Go assay services, this award-winning research partner leverages high-content imaging to enable life scientists to accelerate their discovery of novel therapeutics and improve global health. Pluristyx offers expertise and tools to support and accelerate the development and commercialization of revolutionary therapeutics to treat serious human diseases by providing unique products, consulting, and contract services, including cell line expansion and banking. About Synthego Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms and products for science at scale. With its foundations in engineering disciplines, the company's platform technologies vertically integrate proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance basic research, target validation, and clinical trials. With its technologies cited in hundreds of peer-reviewed publications and utilized by thousands of commercial and academic researchers and therapeutic drug developers, Synthego is at the forefront of innovation enabling the next generation of medicines by delivering genome editing at an unprecedented scale.

Read More


Be The Match BioTherapies® Introduces CIBMTR® CRO Services

Be The Match BioTherapies® | January 18, 2023

On January 17, 2023, Be The Match BioTherapies, a company that provides solutions for organizations that develop and commercialize cell and gene therapies, introduced its offering of CIBMTR® clinical research organization services (CRO services), which significantly reduces the time needed to launch and execute high-impact clinical trials. These CRO services are the outcome of a collaboration between Be The Match BioTherapies and CIBMTR, resulting in expertise and access to unmatched research, sites, donors, partnerships, scientific and operational knowledge, and other resources. CIBMTR CRO services offer unique capabilities such as Established relationships with over 225 clinical locations domestically Nearly 20 years of expertise in transplant and cellular therapy trials and study management, as well as clinically-trained staff On-staff transplant physicians provide direct contact with patients and donors for clinical and research insights. A link to the CIBMTR outcomes database for over 575,000 patients The new CRO services offer turn-key solutions, including protocol development and approval oversight, site selection, project management, site start-up, research sample/laboratory coordination, data management, study monitoring, patient-reported outcomes, statistical analysis, and financial administration. For certain services, such as data analysis, surveys, site selection and administration, and sample management, businesses may instead rely on the expertise of CIBMTR CRO services. About Be The Match BioTherapies® Be The Match BioTherapies is the sole supplier of cell and gene therapy solutions with fully configurable services to assist the cell therapy supply chain. It is committed to accelerating patient access to life-saving cell and gene treatments by delivering the best-quality cellular source material from Be The Match Registry®, the most-varied registry of prospective blood stem cell donors in the world, with over 7 million potential blood stem cell donors. Through global collaborations with apheresis, bone marrow collection, and transplant centers, the firm creates, onboards, trains, and maintains extensive collection networks to advance cell treatments.

Read More


Biocytogen Launches RenNano™ Mouse, a Fully Human Heavy Chain Antibody Platform

Biocytogen | January 05, 2023

RenNano™, a fully human heavy-chain antibody platform developed by Biocytogen Pharmaceuticals Co., Ltd., has been launched officially. RenNano™ joins RenMab™ and RenLite® as the newest member of the RenMice™ family. All three RenMice™ technologies from Biocytogen work together to speed up the search and development of entirely human monoclonal, bispecific/multispecific, and single-domain antibodies (sdAbs, or nanobodies). Camels and sharks produce heavy-chain-only antibodies (HCAb), indicating their variable domains (VHH or sdAb) are functional without pairing with the light chain. Still, humans and mice produce antibodies that require heavy and light chain coupling to be effective. Because of their molecular weight and size, sdAbs can enter solid tumor tissues and breach the blood-brain barrier. Additionally, the sdAb's larger CDR3 region allows for detecting the otherwise concealed epitopes of GPCRs and other challenging targets. Nanobodies offer their benefits, but it's challenging to use animals that naturally make HCAbs, like camels, in large numbers to create monoclonal antibodies due to their size, slow reproduction rate, and limited offspring supply. However, humanizing camelid antibody sequences for pharmaceutical use adds another layer of complexity and time to the drug development process. Accordingly, Biocytogen modified the constant area of the entirely human RenMab™ model to create the RenNano™ Mouse, which is genetically modified to manufacture HCAbs. RenNano™ Mouse is one of the world's most comprehensive human antibody platforms due to the substitution of the mouse genes with the complete human heavy chain variable genes. About Biocytogen Biocytogen, a global biotechnology company, is at the forefront of developing cutting-edge methods for studying and creating new antibody-based pharmaceuticals. Biocytogen has streamlined the entire drug development process by integrating its drug efficacy screening platforms and clinical solid development expertise with its proprietary RenMab™, RenLite®, and RenNano™ platforms for the fully human monoclonal antibody, bispecific/multispecific antibody, and nanobody develo

Read More