CELL AND GENE THERAPY
Cellares Corporation | July 16, 2021
Cellares Corporation, a life sciences technology company that has pioneered a revolutionary automated approach to cell therapy manufacturing announced today that Poseida Therapeutics, Inc., a clinical-stage biopharmaceutical company that uses proprietary genetic engineering platform technologies to create cell and gene therapeutics with the potential to cure, has joined its Early Access Partnership Program (EAPP). Poseida joins PACT Pharma and academic partner Fred Hutchinson Cancer Research Center as the third entity to join Cellares' EAPP.
Cellares launched the EAPP in 2020 to provide participants awareness and early access to Cellares' Cell Shuttle, a next-generation cell therapy manufacturing platform that enables closed, automated, and scalable cell therapy production. Poseida's involvement in the initiative adds to the Cell Shuttle's development, range of usage, and applicability by providing insight and experience in manufacturing processes for various autologous and allogeneic cell therapies.
Poseida is presently testing two autologous CAR-T product candidates in the clinic: P-BCMA-101 for relapsed/refractory multiple myeloma and P-PSMA-101 for metastatic castrate-resistant prostate cancer. The firm, which completed an initial public offering in July 2020, is also developing off-the-shelf versions of these treatments and TCR-T, anti-c-kit CAR-T, induced pluripotent stem cells (iPSCs), genetically modified hematopoietic stem cells (HSCs), and NK cells. In addition, Carl June, M.D., an immunotherapy pioneer and renowned oncologist who advised Cellares on creating the Cell Shuttle has just joined Poseida's Immuno-Oncology Scientific Advisory Board.
Poseida will assess the Cell Shuttle prototypes and give statistics and written comments related to their function and performance as part of Cellares' EAPP. In addition, user studies will be conducted to assess the Cell Shuttle's hardware and software, product requirements, release criteria, and process processes to ensure product-market fit.
About The Cell Shuttle
The Cell Shuttle is a flexible and scalable automated and closed end-to-end production solution that allows clients to execute the precise procedures required for their cell therapy. Compared to presently existing cell therapy manufacturing methods, this next-generation platform allows for a threefold decrease in process failure rates and the ability to produce 10+ patient doses in parallel, improving manufacturing scalability by order of magnitude. For most processes, this will reduce per-patient manufacturing costs by up to 70%.
About Cellares Corporation
Cellares is rethinking cell therapy manufacturing and accelerating access to life-saving cell therapies. The business is working on a one-of-a-kind approach to solving the difficulties of generating cell therapies that are cheaper and broadly accessible to people in need. Cellares' proprietary platform, the Cell Shuttle, eliminates the need for biopharma companies, academic research centers, and CDMOs to choose between a manufacturing platform that is semi-automated but lacks workflow flexibility or one that provides customization but lacks the end-to-end automation required to manufacture at scale. The business is based in South San Francisco, California.
Entos Pharmaceuticals, Inc., BioMarin | November 16, 2021
Entos Pharmaceuticals, Inc. (Entos), a clinical-stage biotechnology company developing genetic medicines with its Fusogenix proteolipid vehicle (PLV) nucleic acid delivery platform, and BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that they have entered into an agreement that will see Entos apply its Fusogenix nucleic acid delivery technology to promising gene therapy candidates in the BioMarin pipeline. Under the agreement, Entos will create specially formulated product candidates for BioMarin, a world leader in developing and commercializing innovative biopharmaceuticals for genetic diseases driven by genetic causes.
Entos' Fusogenix platform utilizes a PLV formulation with a novel mechanism of action to deliver molecular payloads, intact and unmodified, directly into target cells. By combining well-tolerated neutral lipids with proprietary fusion-associated small transmembrane (FAST) proteins, the Fusogenix platform efficiently delivers mRNA or DNA to target tissue with excellent tolerability. Fusogenix technology has been applied to a wide range of therapeutic approaches, including vaccines, gene therapy, and gene editing.
"As a company committed to addressing the unmet therapeutic needs of patients living with genetic diseases, BioMarin values novel technologies that enable the development of transformative therapies," said Brinda Balakrishnan, M.D., Ph.D., Group Vice President, Corporate and Business Development at BioMarin. "We believe that Entos' Fusogenix platform offers potentially unique benefits for safe and effective tissue targeting compared with other lipid-based delivery systems. The Fusogenix PLV formulations generated under this agreement are a critical first step in determining how we may incorporate this promising platform into our drug development efforts."
"With a 20-year track record of successfully discovering, developing, and commercializing first- or best-in-class therapies for diverse rare diseases, the BioMarin team has extensive expertise in harnessing the potential of breakthrough technologies. We believe this licensing agreement helps validate the potential of our Fusogenix platform. It also provides additional opportunities for incorporating our technology into cutting-edge treatments that may have significant clinical and commercial value."
John Lewis, Ph.D., Founder and Chief Executive Officer of Entos
Under the terms of the agreement, Entos will use its Fusogenix nucleic acid delivery system to specially formulate BioMarin products directed at multiple undisclosed genetic disease indications. BioMarin will conduct preclinical studies of the Fusogenix-formulated candidates to evaluate their potential as therapies to prevent or treat these conditions.
Entos' most advanced clinical program is Covigenix VAX-001, a COVID-19 DNA vaccine encoding SARS-CoV-2 Spike protein and two genetic adjuvants engineered to stimulate host adaptive and innate immune systems. Formulated using the Fusogenix platform, Covigenix VAX-001 is the subject of an ongoing phase 2 clinical trial in South Africa, with additional trials planned in other regions. Licensees of the Fusogenix platform include Oisín Biotechnologies and OncoSenX, which are using the technology for age-related diseases and oncology applications, respectively.
pH Partners, LLC served as financial advisor to Entos.
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and preclinical product candidates.
About Entos Pharmaceuticals, Inc.
Entos develops next generation nucleic acid-based therapies using its proprietary Fusogenix proteolipid vehicle (PLV) drug delivery system. Fusogenix is formulated with FAST proteins to deliver mRNA or DNA directly into the cytosol of target cells for translation into protective and therapeutic vaccines and medicines.
GenScript | May 18, 2022
GenScript USA Inc., the world's leading life-science research tools and services provider, announced the availability of new GMP-grade GenExact™ single-stranded DNA (ssDNA) and GenWand™ closed-end linear double-stranded DNA (dsDNA) services. These services will enable next-generation gene and cell therapy R&D programs to advance to IND filing and clinical trials faster and more efficiently.
Today's announcement, made at the American Society of Gene & Cell Therapy (ASGCT) 25th annual meeting (booth #280), further establishes GenScript as the leading provider of non-viral homology-directed repair (HDR) template solutions for immune-cell engineering. GenScript now offers varying grades of non-viral HDR payload materials that support use cases across early discovery research, process development, and clinical studies.
CRISPR-based non-viral gene insertion methods have gained popularity among research teams with concerns about the FDA's recent draft guidance concerning the use of viruses for gene and cell therapy. Non-viral HDR templates, in combination with CRISPR technology, can accelerate cell engineering compared to traditional GMP virus-based engineering, delivering results faster than alternate techniques.
This new investment by GenScript demonstrates our commitment to non-viral delivery technology. We look forward to continuing to work with our partners and stakeholders in academia and industry to support their cell therapy R&D programs and further development of non-viral delivery platforms and workflows, We know this technology will be a more efficient and safer solution for cell engineering."
Ray Chen, PhD, president of GenScript USA Life Science Group.
GenScript's GMP ssDNA And dsDNA services enable faster CAR-T drug development:
GenExact™ ssDNA up to 5000nt in length, 100mg/batch production scale
GenWand™ closed-end, linear dsDNA up to 10,000nt in length, gram level/batch production scale
GenScript has been collaborating with several academic and industry partners in the development of CRISPR non-viral gene insertion. Recently, Dr. Alex Marson's lab at UCSF published a paper demonstrating how CRISPR RNP technology can both knock out endogenous T-cell receptors and integrate replacement anti-cancer receptors.
The UCSF team has been using GenScript's latest single-strand DNA to improve non-viral gene knock-in efficiency. Working in collaboration with GenScript, the team engineered modifications to the DNA payload to promote co-localization and delivery (or 'shuttle') to the nucleus.
About GenScript Biotech Corporation
GenScript Biotech Corporation (Stock Code: 1548.HK) is a global biotechnology group. Based on its leading gene synthesis technology, GenScript has developed four major platforms including the global cell therapy platform, the biologics contract development and manufacturing organization (CDMO) platform, the contract research organization (CRO) platform, and the industrial synthesis product platform.
GenScript was founded in New Jersey, USA in 2002 and listed on the Hong Kong Stock Exchange in 2015. GenScript's business operation spans over 100 countries and regions worldwide, with legal entities located in the USA, mainland China, Hong Kong, Japan, Singapore, the Netherlands, and Ireland. GenScript has provided premium, convenient, and reliable products and services for over 100,000 customers.