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.
Read More
INDUSTRIAL IMPACT, DIAGNOSTICS
Kite Pharma and Arcellx | January 31, 2023
On January 30, 2023, Kite and Arcellx, Inc. informed that they have completed their previously-announced global strategic collaboration to co-develop and co-market CART-ddBCMA.
CART-ddBCMA is Arcellx's lead late-stage candidate for treating patients with relapsed or refractory multiple myeloma.
For the majority of patients, multiple myeloma is an incurable disease; hence, there is a continuing demand for treatments that are not only effective but also secure and easily available.
Arcellx's T-cell treatment, CART-ddBCMA, uses the company's unique synthetic binder, the D-Domain, and is now being studied in Phase 2 pivotal trials. Kite and Arcellx will work together to develop and market the CART-ddBCMA asset in the United States, while Kite will handle sales and marketing for the product in all other regions.
About Kite Pharma
Kite, a Gilead company, is a global biopharmaceutical company with its headquarters in Santa Monica (California). Cell therapy, which aims to treat and maybe even cure cancer, is the company's main area of research and development. Kite, the industry leader in cell therapy, has successfully administered CAR T-cell treatment to more patients than any other company. Process development, vector production, clinical trial supply, and commercial product manufacturing are all part of Kite's expansive in-house cell therapy manufacturing network, the most extensive in the world.
About Arcellx
Clinical biotechnology firm Arcellx, Inc. is rethinking cell treatment by developing novel immunotherapies for patients with cancer and other fatal diseases. The company considers cell therapies one of the most promising areas of modern medicine. Its goal is to improve the quality of life for people everywhere by creating safer, more effective, and widely available cell therapies. CART-ddBCMA is the principal product candidate being developed by Arcellx and is now in Phase 2 pivotal trials for treating relapsed or refractory multiple myeloma (r/r MM). The FDA has recognized CART-potential ddBCMAs by designating them as a regenerative medicine advanced therapy, an orphan drug, and a fast-track product.
Read More
MEDTECH, INDUSTRIAL IMPACT
ATUM and Codiak | January 25, 2023
On January 24, 2023, ATUM, a global leader in bioengineering solutions, announced that it has signed a comprehensive licensing agreement with Codiak BioSciences, a clinical-stage biopharmaceutical company, for the Leap-In Transposase® technology.
ATUM will grant Codiak access to its Leap-In Transposase® technology in order to create mammalian cells expressing exosomes engineered to deliver therapeutic payloads under the conditions of the agreement. The Leap-In technology is a comprehensive solution that integrates patented re-coding algorithms, unique genetic vector elements, and a transposon-based mechanism for efficiently inserting genetically stable elements into the genome.
"Through years of rigorous research and clinical validation we have created a platform for engineering and manufacturing therapeutic exosomes with various payloads on the surface or inside the lumen. We believe ATUM's transposase technology has the potential to effectively synergize with Codiak's engEx® Platform to further enhance loading these vesicles, making them more potent and reducing cost of goods," Commented Scott Estes, Ph.D., VP of Cell Line Development at Codiak.
(Source – Business Wire)
"Exosomes are bio vesicles that have the potential to fundamentally change drug delivery. We are proud and excited to license the Leap-In Transposase® technology to Codiak for the engineering of cell lines that enables rapid and robust production of exosomes," said Claes Gustafsson, Ph.D., Chief Commercial Officer and Co-Founder at ATUM.
(Source – Business Wire)
About Codiak
Founded in 2015, Codiak is a clinical-stage biopharmaceutical firm focused on developing exosome-based treatments, a new class of medications with the potential to change the treatment of a variety of diseases with high unmet medical needs. It created a proprietary engEx Platform that uses exosome biology as natural intercellular transfer mechanisms to design, construct, and manufacture innovative exosome therapeutic candidates. By utilizing its engEx Platform, it has produced a rich pipeline of engineered exosomes for neuro-oncology, oncology, neurology, neuromuscular, and infectious diseases.
Read More