This Week in Cell and Gene Therapy: 11 New Developments to Know (September 12, 2022)

There have been a number of recent developments in cell and gene therapy, as detailed in our bimonthly newsletter, Cell and Gene Therapy Business Outlook.

• Kite Pharma, a Gilead Company based in Santa Monica, CA, has announced that the European Commission (EC) has granted approval for its chimeric antigen receptor (CAR) T cell therapy Tecartus (brexucabtagene autoleucel) for the treatment of adult patients 26 years of age and above with relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukemia (ALL).  Tecartus is an autologous, CAR T cell therapy targeting CD19, an antigen ubiquitously expressed on B cells.  Last year Tecartus was approved by the U.S. FDA for the same indication.
• CellOrigin Biotech, an immune cell therapy company based in Hangzhou, China, and Qilu Pharmaceutical, a pharmaceutical company based in Jinan, China, have announced a collaboration to develop, manufacture, and commercialize off-the-shelf induced pluripotent stem cell (iPSC)-derived chimeric antigen receptor macrophages (CAR-iMAC) for cancer immunotherapy.  CellOrigin’s developmental pipeline currently includes an iPSC-derived natural killer (iNK) + monoclonal antibody therapy candidate for the treatment of hematological malignancies, a CAR-iNK cell therapy candidate for solid tumors, and several CAR-iMAC cell therapy candidates for solid tumors.
• The University of Sydney has announced an unprecedented $478 million investment to build a leading biomedical precinct in New South Wales, its largest ever capital investment. The Sydney Biomedical Accelerator (SBA) will establish a 36,000 square-meter health, education, and research precinct, to be co-located at Royal Prince Alfred Hospital and the University’s Camperdown campuses.  The New South Wales Government contributed $143.3 million to the project, and other funding includes $73 million in philanthropy to the University of Sydney and a $20 million donation from the Susan and Isaac Wakil Foundation to establish The Isaac Wakil Biomedical Building.   The SBA will support over 1200 biomedical researchers and clinician scientists, including over 800 university laboratory researchers and PhD students, with occupation anticipated to begin in 2026.
• Cytiva, a life sciences company based in Marlborough, MA, and Caring Cross, a 501(c)(3) non-profit organization focused on developing advanced medicines accessible to all patients, have announced a partnership to develop a chimeric antigen receptor (CAR)-T cell therapy for people with HIV in low-to-middle-income nations.  Cytiva will provide equipment and software packages, including the Sepax C-Pro, (a place of care instrument which can reduce CAR-T processing times by automatically isolating, concentrating, washing, and diluting cellular products) and the VIA Thaw and VIA Freeze instruments (which streamline freezing and thawing processes while minimizing the contamination risks associated with traditional water baths).  Caring Cross has developed a duoCAR-T cell therapy candidate targeting HIV which can both eliminate HIV-infected cells and protect CD4-T cells from HIV infection in in vitro studies and animal models, and the therapy is currently being evaluated in a Phase I/II clinical trial.  Caring Cross is also developing a stem cell gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia.
• Arbor Biotechnologies, a gene editing company based in Cambridge, MA, has announced that it has entered an agreement with Acuitas Therapeutics, a leader in the development of lipid nanoparticles (LNP) based in Vancouver, BC, to develop therapies for rare liver diseases.  Arbor specializes in discovering and developing novel gene editors using its machine learning/AI driven discovery platform, and has an established pipeline of rare liver disease programs in preclinical development.  The two companies plan to combine Acuitas’ optimized LNP delivery technology with Arbor’s proprietary CRISPR-based in vivo gene editing therapies targeting rare liver diseases to accelerate these programs into the clinic.
• ElevateBio, a cell and gene therapy technology company based in Cambridge, MA, and the University of Pittsburgh have announced a long-term strategic partnership to develop cell and gene therapies.  The 30-year agreement will establish ElevateBio’s next BaseCamp process development and Good Manufacturing Practice (GMP) manufacturing facility at the Pitt BioForge BioManufacturing Center at Hazelwood Green.  The University of Pittsburgh created the Pitt BioForge biomanufacturing facility with a $100 million grant from the Richard King Mellon Foundation in November 2021.  The new BaseCamp facility will be equipped with gene editing and  induced pluripotent stem cell (iPSC) technologies, as well as cell, vector, and protein engineering capabilities, and is expected to generate more than 170 full-time jobs.
• ElevateBio has also announced a partnership with the California Institute for Regenerative Medicine (CIRM) to advance regenerative medicine therapies through CIRM’s Industry Alliance Program.  For its part, ElevateBio will supply its induced pluripotent stem cells (iPSC) lines to academic and commercial beneficiaries of CIRM Discovery and Translational Grants, along with viral vector technology, process and analytical development, and Good Manufacturing Practice (GMP) manufacturing capabilities. CIRM has received $5.5 billion in funding from the state of California, and has already funded over 161 regenerative medicine research projects and 81 clinical trials.
• BioCardia, a cell therapy company based in Sunnyvale, CA, has announced it has entered an agreement with BlueRock Therapeutics, an engineered cell therapy company (and subsidiary of Bayer AG) based in Cambridge, MA, to use BioCardia’s minimally invasive biotherapeutic catheter delivery systems to deliver BlueRock’s induced pluripotent stem cell (iPSC)-derived cell therapy product candidates locally to the heart for the treatment of heart failure. Under the time-limited agreement, BioCardia will receive an up-front payment, and BlueRock will have an option to negotiate a non-exclusive worldwide license to use BioCardia’s biotherapeutic delivery systems to deliver certain cell types for cardiac indications.
• Gensaic, a gene therapy spinout from MIT based in Cambridge, MA, has announced a strategic collaboration agreement with Ovid Therapeutics, a gene therapy company based in New York, NY, to develop up to three genetic medicines for central nervous system (CNS) indications of interest to Ovid using its proprietary phage-derived particle (PDP) platform.  Under the terms of the agreement, Gensaic retains full rights to its PDP platform technology, while Ovid will have the right to license and develop any gene therapies resulting from the collaboration.  Ovid has also invested $5 million in Gensaic and retained rights to invest in future rounds.  Gensaic’s PDP platform is a modular system derived from the M13 bacteriophage (a filamentous virus which infects bacteria) and consists of three engineerable capsid proteins and a minimal phage DNA (mpDNA) genome capable of delivering of genetic payloads larger than 20 kb.  The platform combines phage display technology with directed evolution to engineer tissue-specific, immune-privileged, and redosable delivery vehicles that can be easily manufactured in bacterial culture.  Gensaic is currently focused on developing delivery systems  targeting muscle, respiratory, and CNS tissues.
• Cambridge, MA-based bluebird bio has announced that the U.S. FDA has approved ZYNTEGLO (betibeglogene autotemcel, or beti-cel), its one-time gene therapy for the treatment of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.  Beta-thalassemia is a rare genetic blood disease caused by mutations in the HBB gene encoding β-globin, a major component of hemoglobin A (HbA) resulting in reduced or absent adult hemoglobin production. In Zynteglo therapy, a patient’s hematopoietic stem cells (HSCs) are harvested, then purified and transduced them with a lentiviral vector (LVV) carrying a modified form of the HBB gene encoding β-globin.  The cells are then re-infused back into the patient, where they engraft to the bone marrow and produce new blood cells expressing the modified HBB gene, enabling the cells to make normal or nearly normal levels of adult hemoglobin. The therapy is the first ex vivo lentiviral vector gene therapy to be approved in the U.S. for the treatment of beta-thalassemia. (See Cell and Gene Therapy Business Outlook vol. 2, issue 1, p. 13 for more on bluebird bio and Zynteglo, its gene therapy for beta-thalassemia.)    
• GentiBio, a biotherapeutics company based in Boston, MA, has announced that it has entered a collaboration with Bristol Myers Squibb, based in New York, NY, to develop engineered regulatory T (Treg) cell therapies for the treatment of patients with inflammatory bowel diseases (IBD).  GentiBio specializes in engineered Treg cell therapies, which have the potential to treat autoimmune and inflammatory diseases such as IBD by re-establishing immune tolerance in a tissue-specific manner.  Under the multi-year collaboration, GentiBio will produce stable, disease-specific engineered Tregs against multiple targets utilizing its modular engineered Treg platform and scalable manufacturing process, and Bristol Myers Squibb will have the right to bring up to three programs resulting from the collaboration into the clinic.  In exchange, Bristol Myers Squibb has made an undisclosed cash payment to GentiBio up front, and GentiBio is eligible to receive up to $1.9 billion in development and sales milestones, plus royalties.