AI, INDUSTRIAL IMPACT
Cimeio | December 12, 2022
The two studies demonstrate proof-of-concept that genetically engineered variants of cell surface receptors harboring single amino acid substitutions are fully functional but evade a paired immunotherapy directed against the wild-type molecule. This technology will enable the development of more efficient and safer targeted conditioning regimens for hematopoietic stem cell transplantation, gene therapies, and opens the path to more efficient cell and immunotherapy-based treatment approaches for hematological malignancies.
The first abstract was titled “Function-preserving single amino acid substitutions shield hematopoietic stem and progenitor cells from CD117 targeted immunotherapy in vivo.” The receptor tyrosine kinase c-KIT is expressed on normal hematopoietic stem cells but also on leukemia cells. Consequently, it is an attractive target for an antibody-based conditioning therapy.
The study showed that cells expressing a genetically engineered variant of CD117 are fully functional in vitro and contributed to the blood development in vivo, similar to unmodified HSCs expressing the wt receptor. Mice transplanted with a mix of human HSCs expressing either wt CD117 or the Cimeio shielded CD117 variant showed a selective depletion of wt CD117 cells, while those cells expressing the variant receptor were spared following the treatment with a monoclonal antibody directed against wt CD117.
The second abstract, titled “Engineered Single Amino Acid Substitutions Protect Hematopoietic Stem and Progenitor Cells From CD123 Targeted Immunotherapy,” focused on targeting the interleukin-3 receptor alpha-chain a cytokine receptor highly expressed on various hematological malignancies as well as normal hematopoietic cells. This abstract demonstrated that shielded CD123 cells were not depleted by a highly efficient CD123 targeted immunotherapy.
Collectively, these preclinical studies demonstrate the feasibility and efficacy of Cimeio’s SCIP platform, a novel and promising therapeutic approach to improve the outcomes for patients with benign and malignant hematological diseases in need of an HSC transplant.
“Many severe and late side effects following an HSC transplant are caused by untargeted and broadly toxic chemotherapeutics and radiation in conditioning regimens. The presented work is an encouraging and important step towards safer and efficient approaches to overcome these hurdles. Cimeio’s SCIP platform could allow more patients facing debilitating and fatal diseases to receive a life-saving HSC transplant.”
Suneet Agarwal, M.D., Ph.D., Co-Program Leader for the Stem Cell Transplant Center at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Associate Professor of Pediatrics at the Harvard Medical School, and member of the Cimeio Scientific Advisory Board
Last month, Cimeio announced the issuance of a foundational patent that covers a method for in vivo selective depletion of edited primary hematopoietic cells or non-edited primary hematopoietic cells, the basis of its SCIP platform.
Cimeio is an applied gene editing and immunotherapy company developing a portfolio of Shielded-Cell & Immunotherapy Pairs™ which has the potential to transform hematopoietic stem cell transplant. Cimeio’s technology platform is based on the design and expression of modified variants of naturally occurring cell surface proteins in HSCs. These novel variants maintain their function but are resistant to depletion when targeted by a paired immunotherapy which has high affinity for the wild-type version of these proteins. This technology has significant therapeutic potential, which Cimeio is using to develop curative treatments for patients with genetic diseases, hematologic malignancies, and severe autoimmune disorders.
CELL AND GENE THERAPY, MEDICAL
Sanisure | December 21, 2022
Sanisure, a leading, vertically-integrated player in the single- use bioprocessing technology space, is pleased to announce the acquisition of Medical Elastomer Development, Inc a silicone extrusion facility from Q Holding, a 3i portfolio company. This state-of-the-art facility located in Twinsburg, Ohio, serves many industry- leading customers in both the medical device and bioprocessing markets.
The acquisition further advances our commitment to become a leading provider of critical fluid path management technologies for bioprocessing applications, increasing security of supply and change control for our single use assembly solutions and transfer sets. Following the acquisition, we will be able to offer our fully qualified, high- quality bulk tubing to end-users, other OEMs and our channel partner distributors.
With vertically integrated manufacturing and assembly operations in both North America and Europe, Sanisure offers its customers a wide range of capabilities on a global basis. Sanisure offers leading solutions for critical fluid path technologies, including Mixed4Sure™ closed Carboy Mixing system, Cap2v8® solutions, PharmaTainer™ bottles & carboys, aSURE® fittings, Bio-Ease™ clamps, Gamma stable Stir Bars and an increased range of silicone extrusion and other elastomeric solutions such as the Cellgyn® TPE, TPFE, Kynar tubing, etc.
"We are excited to have the MED Twinsburg Team joining Sanisure's growing family. We continue to invest in critical technologies and capabilities that drive innovation and supply security for our global customer base. State-of- the-art silicone extrusion capabilities are core to our portfolio of fluid management products and solutions, and enable us to provide both high quality silicone tubing and vertically integrated bioprocessing single-use solutions to our customers."
Steven Chevillotte, CEO of Sanisure
Commenting on the acquisition, 3i Partner Richard Relyea said: "We continue to support Sanisure's strategy through both organic and inorganic growth opportunities. This addition further strengthens Sanisure's offering to key customers working on the development and commercialization of monoclonal antibody, vaccine, and cell and gene therapy modalities with single-use technologies in a variety of upstream and downstream applications."
Sanisure designs, develops, and manufactures single-use solutions for the bioprocessing industry including customized bottle assemblies, low-volume mixing solutions aseptic transfer systems, molded caps, flasks, tubes, and clamps. The business now operates six facilities in North America and Europe. Sanisure has longstanding relationships with leading biopharma and biotech Customers, exhibiting an impressive reputation for high-quality solutions and Customer service. In addition to its assembly offerings, Sanisure provides Customers several unique, IP-backed manufactured solutions such as Mixed4Sure™ and Cap2V8™ to address our customers' most demanding applications.
INDUSTRIAL IMPACT, MEDICAL
Inscripta, Inc. | January 24, 2023
Inscripta®, a leading global life science technology firm, recently announced the acquisition of two synthetic biology pioneers, Infinome Biosciences and Sestina Bio, which will advance Inscripta's strategy of developing and commercializing biomanufactured products for a wide expanse of industrial and consumer markets.
The acquisitions show Inscripta's belief in the bioeconomy's commercial potential and the power of genome engineering to expedite biomanufacturing innovation. Inscripta's CEO, Sri Kosaraju, commented, "Tackling the unprecedented opportunity to improve global health and sustainability through biomanufacturing requires a strong combination of talent, experience, technology, and commercial assets." He added, "Infinome and Sestina Bio allow us to advance our strategy and complement our technological innovation and capabilities needed for creating the next generation of biobased manufacturing products and processes."
(Source- PR Newswire)
Before the acquisition, Infinome was closely associated with Inscripta. By utilizing Inscripta's genome engineering technology, it created the GenoScaler™, a proprietary strain engineering platform designed to optimize microorganisms for biomanufacturing rapidly.
Sestina Bio brings an innovative, data-driven approach to developing and identifying strains that survive the rigors of commercial scale-up.
Inscripta is building on its tradition of technological innovation with the inclusion of Infinome and Sestina Bio and has already begun applying its cutting-edge technology to develop products for a diverse set of industrial and consumer markets. In addition, Inscripta has a number of collaborative and proprietary products in various phases of development. One such example of an announced partnership to develop and commercialize multiple bio-manufactured goods is Kalsec® Inc., a food and beverage ingredient solutions firm. Separately, Inscripta is engaging in commercial discussions about its previously announced development of a ready-to-scale microbial strain that produces Bakuchiol, a natural retinol alternative.
About Inscripta, Inc.
Founded in 2015, Inscripta is a life science technology firm dedicated to using biomanufacturing to create a cleaner, healthier, and more sustainable world. It enables scientists to strengthen and expand domestic biomanufacturing and encourages the development of novel, safe, and secure biotechnology technologies. Its automated OnyxTM platform, which includes an instrument, consumables, tests, and software, makes CRISPR-based genome engineering accessible to any research lab. Inscripta supports customers globally through its facilities in Boulder (Colorado), San Diego and Pleasanton (California) and Copenhagen (Denmark)