CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Sana Biotechnology | January 30, 2023
Sana Biotechnology, Inc., a firm focused on producing and delivering engineered cells as medicines, recently announced that the FDA has approved the company's Investigational New Drug (IND) application to begin a first-in-human trial of SC291 in patients with different types of B-cell malignancies.
SC291 is an allogeneic CAR T cell therapy that targets CD19. It is developed by Sana using its hypoimmune platform. The hypoimmune platform's objective is to overcome the allogeneic cells' immunologic rejection, which, if valid for SC291, could lead to prolonged CAR T cell persistence and increased rates of durable complete responses in patients with B-cell lymphomas or leukemias.
The hypoimmune platform includes disruption of the major histocompatibility (MHC) class I and class II expression to conceal cells from the adaptive immune system, which consists of antibody and T cell responses and overexpression of CD47 to inhibit activation of the innate immune cell system, especially macrophages and natural killer (NK) cells. The company has provided evidence from numerous preclinical models demonstrating the potential of this platform to cloak cells from immune detection and the therapeutic potential of SC291 for patients with B-cell malignancies.
About Sana Biotechnology
Founded in 2018, Sana Biotechnology is a firm focused on utilizing engineered cells as medicines for patients. It is constructing differentiated capabilities across the spectrum of cell and gene therapy. The company is motivated by three goals as it seeks solutions for people with poor outcomes or diseases that are currently incurable. The first is the ability to repair and control the genes in any cell in the body. It is developing innovative delivery technologies in order to deliver any payload to any cell in a precise, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next comes the ability to develop pluripotent stem cells ex vivo into immune-cloaked functional cells, to replace any missing or damaged cells in the body. Finally, it believes that it can also enable broader access to its therapies by focusing on scalable manufacturing solutions, the manufacturing costs, and aligning with key stakeholders.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Lineage Cell Therapeutics, Inc. | February 23, 2023
On February 22, 2023, Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology firm focused on the development of allogeneic cell therapies to restore and replace specific cell types of the human body, announced that it has entered into an exclusive option and license agreement with Eterna Therapeutics Inc. (Eterna) for the development of innovative beta 2 microglobulin (B2M)-deficient induced pluripotent stem cell (iPSC) lines that Lineage will assess for the creation of unique, differentiated cell transplant therapies.
These new cell lines to be developed by Eterna are expected to help Lineage generate more product candidates for the treatment of central nervous system (CNS) conditions and other neurology indications. Eterna holds the exclusive license for the crucial intellectual property associated with this partnership, which it obtained from its discovery partner Factor Bioscience.
Following the agreement, Eterna intends to perform specific gene-editing activities and furnish materials to Lineage for assessment. In addition, the agreement grants Lineage the option to obtain an exclusive license to use and sublicense the new gene-edited cell lines for clinical, preclinical, and commercial purposes in the field of CNS diseases. A distinctive feature of the starting cell line is the targeted elimination of the B2M gene, which is aimed at minimizing the immunogenicity of product candidates generated from the cell lines by inhibiting rejection by CD8+ T cells.
Lineage anticipates that this attribute will enhance the overall usefulness of the edited cell lines, including non-immune privileged or non-human leukocyte antigen (HLA) matched indications. Other proposed gene modifications may additionally differentiate the cell line from those currently utilized by competitors. No details of the financial agreement were disclosed.
About Lineage Cell Therapeutics, Inc.
Founded in 1990, Lineage Cell Therapeutics is a clinical-stage biotechnology company focused on developing novel cell therapies to address unmet medical needs. The company's programs are based on its proprietary cell-based therapy platform and related development and manufacturing capabilities. By using this platform, it specializes in the development and manufacture of specialized, terminally-differentiated human cells from its progenitor and pluripotent cell starting materials. These differentiated cells are intended to either support or replace cells that are dysfunctional or absent due to degenerative disease or traumatic injury and can also be administered to help the body mount an effective immune response to cancer.
INDUSTRIAL IMPACT, MEDICAL
Accesswire | March 24, 2023
Moderna, Inc. a biotechnology company pioneering messenger RNA therapeutics and vaccines, and Generation Bio Co. a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that the two companies have entered into a strategic collaboration to combine Moderna's biological and technical expertise with core technologies of Generation Bio's non-viral genetic medicine platform. The collaboration aims to expand the application of each company's platform by developing novel nucleic acid therapeutics, including those capable of reaching immune cells, to accelerate their respective pipelines of non-viral genetic medicines.
"Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients," said Rose Loughlin, Ph.D., Moderna's Senior Vice President for Research and Early Development. "Through this collaboration, which builds on Generation Bio's non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement. We are excited to have Generation Bio as our partner as we continue to broaden our therapeutic pipeline and extend the potential benefit of nucleic acid therapeutics to more patients."
"Non-viral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale," said Phillip Samayoa, Ph.D., Chief Strategy Officer of Generation Bio. "This collaboration represents a foundational investment in our platform science, both deepening our pipeline of rare and prevalent liver disease programs beyond hemophilia A and accelerating our work to reach outside of the liver with nucleic acid therapies. We are thrilled to collaborate with Moderna to extend genetic medicines to new tissues and cell types through the joint development of novel targeting for our stealth ctLNPs to reach immune cells."
About the Collaboration
Under the terms of the agreement, Moderna may advance two immune cell programs, each of which may use a jointly developed ctLNP to deliver ceDNA. In addition, Moderna may advance two liver programs, each of which may use a liver-targeted ctLNP developed by Generation Bio to deliver ceDNA. Moderna retains an option to license a third program for either immune cells or the liver.
Generation Bio will receive a $40 million upfront cash payment and a $36 million equity investment issued at a premium over recent share prices. Moderna will fund all collaboration work, including a research pre-payment. Generation Bio is also eligible for future development, regulatory and commercial milestone payments, as well as royalties on global net sales of liver-targeted and immune cell-targeted products commercialized under the agreement. The agreement additionally provides Moderna with the right, subject to certain terms and conditions, to purchase additional shares of common stock in connection with a future equity financing by Generation Bio.
Further, Moderna and Generation Bio will both leverage collaboration research to continue to advance in vivo immune cell targeting as a new class of genetic medicines, with downstream economics on products utilizing such technology. Generation Bio is eligible to receive certain exclusivity fees as well as potential development and regulatory milestones and royalties on products that Moderna advances using ctLNP technology developed under the collaboration.
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years.