CELL AND GENE THERAPY, MEDICAL
Asher Bio | December 13, 2022
Asher Biotherapeutics, Inc. a biotechnology company focused on developing therapies to precisely engage specific immune cells to fight cancer, today announced new preclinical data demonstrating proof-of-concept for cis-targeted cytokines as a novel strategy for enhancing chimeric antigen receptor T cell engraftment, expansion, and functionality culminating in the delivery of improved anti-tumor activity. This data will be presented in a poster at the 64th American Society of Hematology Annual Meeting being held in New Orleans, Louisiana, December 10-13, 2022.
“We are delighted to share new preclinical data with our cell therapy program, further highlighting the versatility of our cis-targeting cytokine platform, as well as its potential applicability across a range of hematologic malignancies and solid tumors. These data demonstrate our ability to selectively activate only CAR-T cells post-adoptive transfer and, as a result, to improve anti-tumor activity. We are particularly pleased to observe these results using two different cytokines, interleukin-2 and interleukin-21 which act through distinct, potentially complementary, mechanisms. Over time, our ability to deliver different, supportive cytokine signals could enable us to target a diverse set of cell therapies, including those based on T cell receptors, tumor-infiltrating lymphocytes, natural killer cells and regulatory T cells, to support the use of cell therapies in a diverse set of tumor types. We look forward to advancing this program as we aim to maximize the reach of our platform, with the goal of delivering better outcomes to cancer patients who remain underserved by existing therapeutic options.”
Ivana Djuretic, Ph.D., Founder and Chief Scientific Officer of Asher Bio
CAR-T cell therapies have recently transformed the treatment of some hematological malignancies and are showing promise in solid tumors. In the clinic, the successful expansion and persistence of CAR-T cells has correlated with improved therapeutic outcomes, including durable complete responses and survival; in preclinical studies, the administration of IL-2 has been observed to enhance CAR-T engraftment, persistence and functionality. However, the clinical potential of utilizing existing IL-2 molecules in combination with CAR-T therapies is limited due to the severe toxicity of high dose IL-2 and the inadequate selectivity of existing engineered IL-2 variants, which expand multiple endogenous cell types in addition to the transferred CAR-T cells.
In order to overcome these challenges, Asher Bio developed two cis-targeted fusion molecules, one using IL-2 and the other using IL-21, which are designed to selectively activate CAR-T cells by recognizing an extracellular tag, while exhibiting minimal effects on non-engineered or endogenous cells. Both cis-targeted cytokine fusions are comprised of a targeting antibody directed against a tag expressed on the CAR-T surface, that is co-expressed with the CAR, and a cytokine mutein with attenuated binding to its cognate cytokine receptor.
About Asher Bio
Asher Bio is a biotechnology company developing therapies to precisely engage specific immune cells to fight cancer, chronic viral infection, and autoimmune disease. We utilize our proprietary cis-targeting platform to develop therapies engineered to overcome limitations of other immune-based treatments by selectively activating specific immune cell types with validated disease fighting functionality. Our candidates feature an antibody connected to a modified immunomodulatory protein, such as a cytokine. Our candidate design is intended to enable our candidates to selectively activate the desired immune cells and not other cells that contribute to toxicity or immune suppression. Asher Bio was founded by Ivana Djuretic and Andy Yeung with support from Third Rock Ventures and is located in South San Francisco.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Vertex | December 09, 2022
Vertex Pharmaceuticals Incorporated and Entrada Therapeutics, Inc. announced a global collaboration focused on discovering and developing intracellular Endosomal Escape Vehicle therapeutics for myotonic dystrophy type 1. The collaboration includes Entrada’s program for DM1, ENTR-701, which is in late-stage preclinical development.
“Vertex’s strategy is to discover and develop transformative medicines for people with serious diseases, and DM1 has therefore been a disease area of interest to Vertex for some time. Entrada’s innovative EEV approach, the significant progress in their DM1 program, and the potential for it to reach the clinic in the near-term hold exciting potential for patients. Working together, we believe we have the opportunity to develop a transformative treatment for this devastating disease.”
David Altshuler, M.D., Ph.D., Executive Vice President, Global Research, and Chief Scientific Officer of Vertex
“Our collaboration with Vertex represents an important step for Entrada as we work to make intracellular therapeutics a reality through our novel EEV approach,” said Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics. “DM1 is a progressive disease with no treatment options available. Working with Vertex will enable us to expeditiously move this program forward, while focusing the majority of our internal resources on advancing new therapeutic options for patients living with Duchenne and expanding our commitment to non-neuromuscular disease programs.”
The agreement includes a four-year global research collaboration whereby Entrada will continue to advance and receive payments for certain research activities related to ENTR-701, as well as additional DM1-related research activities. Vertex will be responsible for global development, manufacturing and commercialization of ENTR-701 and any additional programs stemming from Entrada’s DM1 research efforts.
About ENTR-701
ENTR-701, a proprietary Endosomal Escape Vehicle conjugated phosphorodiamidate morpholino oligomer, is the second novel clinical candidate from Entrada’s growing pipeline of EEV therapeutics. ENTR-701 is designed to address the underlying cause of myotonic dystrophy type 1 through allele-specific targeting of the disease-associated trinucleotide repeats in dystrophia myotonica protein kinase transcripts. In doing so, ENTR-701 has the potential to restore the function of muscle blind-like proteins, correct the mis-splicing and aberrant expression of downstream transcripts and restore normal muscle function. Data from preclinical studies of ENTR-701 suggest correction of disease relevant biomarkers in various muscle groups.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes and alpha-1 antitrypsin deficiency.
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INDUSTRIAL IMPACT, MEDICAL
Celularity | January 17, 2023
Celularity Inc., a biotechnology firm based in the United States that develops placental-derived allogenic cell treatments and biomaterials, and CH Trading Group LLC, an international import, export, and trading corporation, have agreed to an exclusive territorial distribution agreement.
CH Trading Group will serve as the exclusive territorial distributor for halal-certified products previously announced by Celularity in over 100 countries. The product distribution agreement for halal-certified products by Celularity provides a five-year minimum accumulated purchase commitment of US $225 million.
CEO of CH Trading Group, Sayed Zayan, said, “Based on market demand for Celularity’s commercial biomaterial products, we believe there is a billion-dollar commercial opportunity across the Middle East and North Africa Islamic markets.” He also added, “We are excited to be in a position to expand access to these important medicines and wellness products to more people across the region through our exclusive territories distributor agreement with Celularity.”
(Source – Business Wire)
“According to Emergent Research's most recent analysis, the global regenerative medicine market was valued at $9.80 billion in 2021 and is predicted to expand at a CAGR of 15.9%, reaching $37.10 billion in 2030.”
Under the agreement, CH Trading Group will distribute products to more than 100 countries that are members of or affiliated with the following intergovernmental organizations (‘Islamic Markets’)
The Gulf Cooperation Council: Consists of Bahrain, Oman, Kuwait, Qatar, Saudi Arabia, and the UAE.
The Organization of Islamic Cooperation: It spans four continents and includes 57 member nations
The African Union: Includes 55 member countries
Robert J. Hariri, M.D., Ph.D., Celularity’s CEO, Chairman and Founder, said, “Our partnership with the CH Group represents another critical milestone towards bringing our innovative halal-certified—under globally recognized Circle H International Inc. standards – regenerative biomaterial products to treat degenerative disease across Islamic Markets.”
(Source – Business Wire)
About Celularity
Headquartered in Florham Park, N.J., Celularity is a clinical-stage biotechnology firm paving the way for the future of cellular medicine by offering off-the-shelf allogeneic placenta-derived cellular therapeutics with unmatched scale, quality, and affordability. With its novel approach to cell therapy, the company can unlock the unique therapeutic potential of the postpartum placenta. In addition, through the placenta, nature’s immunotherapy engine, the firm is driving the next generation of cellular medicine using placenta-derived T cells, NK cells, and pluripotent stem cells to address unmet and underserved clinical needs in cancer, infectious, and degenerative diseases.
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