Contributing Author: Gina Hagler
Maryland has one of the largest life science clusters in the United States with more than 500 biotech companies. In addition to several state-of-the-art labs, Maryland is home to the NIH in Bethesda. Research opportunities, as well as BS and MS degree programs, are offered at Johns Hopkins in Baltimore and the University of Maryland in College Park. Montgomery College in Germantown offers the AAS degree and certificate programs in biotech. World-class training by companies like BioTrac and Biotech Primer attracts an international audience of professionals.
Maryland’s biopharma companies working with gene and cell therapies are revolutionizing drug discovery and drug development as they create the next generation of medicine. These biopharmaceutical companies make use of a variety of healthy cell types to develop therapies to repair or replace damaged cells in a patient’s body. Here is the latest news from seven of these cell therapy companies.
In February 2020, WindMil announced a collaborative pre-clinical research partnership with the University of Pennsylvania (Penn) to explore novel chimeric antigen receptor (CAR)-engineered MILs (CAR-MILs™) in hematological and solid tumor model settings. The collaboration will leverage WindMIL’s and Penn’s respective expertise to conduct pre-clinical comparisons of the characteristics and functionality of CAR-T and CAR-MIL products.
WindMil Therapeutics is a private clinical-stage company developing a novel class of autologous cell therapies based on marrow-infiltrating lymphocytes (MILs™) for cancer immunotherapy. WindMIL uses its novel insights in bone marrow immunology to create life-saving cancer immunotherapeutics for patients. Their proprietary process activates and expands these cells and offers immunotherapeutic advantages that are unique. These advantages include inherent tumor-specificity, high cytotoxic potential, and long persistence.
Product: Unmodified MILs and CAR-MILs
Long-Term: To translate novel insights in bone marrow immunology into life-saving cell therapies for cancer patients.
In June 2020, MaxCyte, the global clinical-stage cell-based medicines and life sciences company, announced the launch of the first product in the new and expanded range of ExPERT™ disposables. The new R-1000 cuvette can process a volume of up to 1 mL, or up to 200 million cells, and provides increased versatility for companies developing cell therapy drugs as well as those advancing early drug discovery. This expands the company’s range of disposables and provides additional growth opportunities by addressing one of the processing volumes frequently requested by customers. Based on extensive customer feedback, this new design can be used across the complete range of MaxCyte’s electroporation instrumentation, including the recently launched ExPERT ATx™, STx™ and GTx™, and represents the first product in a new industrial design that provides improved usability and handling.
Maxcyte is focused on accelerating the discovery, development, and manufacturing of cell-based medicines to address a range of acute and chronic diseases. Their partners in biopharma will create new cell therapy medicines through the use of MaxCyte’s ExPERT platform. MaxCyte also has a proprietary mRNA-based therapeutic platform for autologous cell therapy. These CARMA therapies can be used to create treatments for solid cancers.
Symbol: MXCT (NASDAQ)
Product: Flow Electroporation® Technology, ExPERT Platform
Long-Term: To accelerate the development of cell-based treatments such as small molecule drugs, biologics, vaccines, and cell and gene therapies to address a range of acute and chronic diseases.
In October 2020, Precigen announced US Food and Drug Administration (FDA) clearance and successful technology transfer for its UltraPorator™ system, an exclusive device and proprietary software solution for the scale-up of rapid and cost-effective manufacturing of UltraCAR-T® therapies. The FDA cleared UltraPorator as a manufacturing device for clinical trials of Precigen's investigational UltraCAR-T therapies in compliance with current good manufacturing practices (cGMP). In addition, the team has successfully completed technology transfer of the UltraPorator system for the manufacturing of UltraCAR-T in the ongoing clinical trials for PRGN-3005 in ovarian cancer at the University of Washington/Fred Hutchinson Cancer Research Center and for PRGN-3006 in acute myeloid leukemia (AML) at the Moffitt Cancer Center.
Precigen’s approach to the design of potent on-target gene and cellular multifunctional therapies is accomplished through the use of their design approach. This disciplined approach uses viral and non-viral suppression systems, genome, DNA, RNA, and protein engineering, with a suite of precision bioengineering switch technologies to control gene expression and regulation to deliver therapies with improved safety and efficacy.
Symbol: PGEN (NASDAQ)
Product: PRGN-3009, PRGN-3010, PRGN-2012
Long-Term: To discover, develop, and commercialize next-generation gene and cell therapies focused in immuno-oncology, autoimmune disorders, and infectious diseases.
In October 2020, NexImmune announced that it had signed a research initiative related to its AIM nanoparticle technology with City of Hope, a world-renowned independent research and treatment center for cancer, diabetes, and other life-threatening diseases. City of Hope is a participating clinical site in the ongoing Phase 1/2 study of NEXI-001. The cancer center will leverage both patient samples from the ongoing NexImmune Phase 1/2 clinical study of NEXI-001 in acute myeloid leukemia (AML) patients with relapsed disease after allogeneic stem cell transplantation and the center’s tumor repository bank of primary leukemia samples, one of the largest collections in the world, to drive the research. NEXI-001 is a cellular product candidate that contains populations of naturally occurring CD8+ T cells directed against multiple antigen targets for AML, and it is the first clinical product generated by the company’s AIM nanoparticle technology.
NexImmune is a private clinical-stage biotechnology company. The company develops unique non-genetically-engineered T cell immunotherapies through the use of its Artificial Immune Modulation (AIM) proprietary technology. The modular design of the AIM platform enables rapid expansion across multiple therapeutic areas, with both cell therapy and injectable products. NexImmune’s pipeline has preclinical programs, including cell therapy and injectable product candidates, for the treatment of oncology, autoimmune disorders, and infectious diseases.
Product: NEXI-001, NEXI-002, NEXI-003
Long-Term: To harness the power of precision technology to restore natural immunity in the treatment of life-threatening diseases.
In November 2020, AGT announced that the first trial participant was enrolled in the Phase 1 trial of AGT103-T, a new cell and gene therapy for HIV disease. The first trial participant underwent leukapheresis, a procedure for obtaining concentrated white blood cells, which will be used for manufacturing the AGT103-T cell product. After manufacturing and required safety testing, the product will be infused back into the trial participant to test the safety and its effect on HIV. Phase 1 is in the Maryland/DC Area.
American Gene Technologies is a private biotechnology company dedicated to finding cures and treatments for infectious diseases, cancers, and monogenic disorders. AGT uses cell and gene therapy in its work. Their current research programs use modified T cells for a prospective functional HIV cure, lentiviral based gene therapy for a PKU cure, and immuno-oncology program that targets cancerous solid tumors.
Technology: Rapid lentiviral vector gene delivery platform
Long-Term: To leverage the power of gene and cell therapy to reduce human suffering or early death from serious human diseases.
In June 2020, Autolus Therapeutics announced preclinical data related to AUTO5 in T cell lymphoma and AUTO6NG in small cell lung cancer, as well as an oral presentation related to AUTO7 in prostate cancer at the American Association for Cancer Research (AACR) Virtual Annual Meeting II on June 22 - 24, 2020. “Behind our lead programs AUTO1 in ALL and AUTO3 in DLBCL, we have a number of exciting preclinical product candidates progressing towards the clinic,” said Dr. Christian Itin, chairman and chief executive officer of Autolus. “These data updates for AUTO5, AUTO6NG, and AUTO7 illustrate the strength of our broad and modular cell programming technology to adapt the product properties to the specific tumor type.”
Autolus Therapeutics is a clinical-stage biopharmaceutical company founded on advanced cell programming technology. It is a leader in T cell programming and manufacturing technology. The company works with physicians and other healthcare providers to extract immune cells from patients, equip them with a receptor that targets the cancer cell, and infuse the patient’s modified cells back into the patient. Autolus believes its immuno-oncology therapies will offer cancer patients clear benefits over existing treatments.
Symbol: AUTL (NASDAQ)
Long-Term: To bring life-changing treatments to cancer patients by reprogramming their own T cells to combat hematological malignancies and solid tumors.
In November 2020, Arcellx announced that clinical data from its ongoing Phase 1 Study of CART-ddBCMA, a genetically engineered cell therapy utilizing the company’s novel synthetic binding domain for the treatment of patients with relapsed and refractory multiple myeloma, will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting, taking place virtually Dec. 5-8, 2020. CART-ddBCMA is a Phase 1 study of Arcellx’s BCMA-specific CAR-modified T-cell therapy utilizing the company’s novel BCMA-targeting binding domain for the treatment of patients with relapsed and refractory multiple myeloma
Arcellx is a private clinical-stage biopharmaceutical company. It develops noble, adaptive, and controllable cell therapies for the treatment of patients with cancer and autoimmune diseases. Arcellx’s paper, “Chimeric Antigen Receptors Incorporating Novel (non-scFv) Binding Domains Targeting CD123 Direct Potent Antitumor Activity of T Cells: Correlation Between Affinity and Activity” was presented at the American Association for Cancer Research (AACR) 2020 Virtual Annual Meeting II.
Product: CAR T-Cell Therapy for the treatment of cancer
Long-Term: To bring ARC-sparX platform cell therapies to millions of patients who can self-administer prescribed sparX proteins under the care of academic and community practices.
In September 2020, RoosterBio announced it had entered into a Cooperative Research and Development Agreement (CRADA) with The Geneva Foundation (Geneva), a non-profit dedicated to advancing military medicine, and the Uniformed Services University of the Health Sciences (USU), an institution of higher learning within the United States Department of Defense (DoD) on behalf of USU’s 4 Defense Biotechnology, Biomanufacturing, and Bioprinting Center (4D Bio3). Under the terms of the CRADA, RoosterBio will support biofabrication in austere environments and provide subject matter expertise in Ready to Print (RTP) technologies.
RoosterBio is radically simplifying the use of adult human mesenchymal stem/stromal cells (hMSCs) in order to propel the commercialization of regenerative technologies. Their living cellular technologies are more affordable, easier to access, and much simpler to incorporate into product development efforts than the current time-consuming and costly technologies. This will lead to a rapid acceleration in products that incorporate these technologies coming to market.
Product: an industrialized supply chain of high-quality hMSCs
Long-Term: To incorporate MSCs into technology pipelines to drive a tremendously positive impact on human healthcare.