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)
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.
Rocket Pharmaceuticals, Inc. | December 02, 2022
Rocket Pharmaceuticals, Inc. a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that it has completed the previously announced acquisition of Renovacor, Inc. a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. Under the terms of the merger agreement entered into on September 19, 2022, Renovacor shareholders received, for each share of Renovacor common stock, 0.1763 shares of Rocket common stock.
The acquisition provides Rocket with Renovacor’s most advanced program, REN-001, an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. Additionally, Rocket gains access to world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities altogether further extend Rocket’s leadership position in AAV-based cardiac gene therapy and help advance the Company’s goal of pursuing gene therapy cures for patients living with rare and devastating diseases.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has a preclinical AAV-based gene therapy program in BAG3-associated dilated cardiomyopathy.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Biocytogen | January 05, 2023
RenNano™, a fully human heavy-chain antibody platform developed by Biocytogen Pharmaceuticals Co., Ltd., has been launched officially. RenNano™ joins RenMab™ and RenLite® as the newest member of the RenMice™ family. All three RenMice™ technologies from Biocytogen work together to speed up the search and development of entirely human monoclonal, bispecific/multispecific, and single-domain antibodies (sdAbs, or nanobodies).
Camels and sharks produce heavy-chain-only antibodies (HCAb), indicating their variable domains (VHH or sdAb) are functional without pairing with the light chain. Still, humans and mice produce antibodies that require heavy and light chain coupling to be effective. Because of their molecular weight and size, sdAbs can enter solid tumor tissues and breach the blood-brain barrier. Additionally, the sdAb's larger CDR3 region allows for detecting the otherwise concealed epitopes of GPCRs and other challenging targets.
Nanobodies offer their benefits, but it's challenging to use animals that naturally make HCAbs, like camels, in large numbers to create monoclonal antibodies due to their size, slow reproduction rate, and limited offspring supply. However, humanizing camelid antibody sequences for pharmaceutical use adds another layer of complexity and time to the drug development process.
Accordingly, Biocytogen modified the constant area of the entirely human RenMab™ model to create the RenNano™ Mouse, which is genetically modified to manufacture HCAbs. RenNano™ Mouse is one of the world's most comprehensive human antibody platforms due to the substitution of the mouse genes with the complete human heavy chain variable genes.
Biocytogen, a global biotechnology company, is at the forefront of developing cutting-edge methods for studying and creating new antibody-based pharmaceuticals. Biocytogen has streamlined the entire drug development process by integrating its drug efficacy screening platforms and clinical solid development expertise with its proprietary RenMab™, RenLite®, and RenNano™ platforms for the fully human monoclonal antibody, bispecific/multispecific antibody, and nanobody develo