Globenewswire | May 29, 2023
Monte Rosa Therapeutics, Inc. a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, announced its second development candidate, MRT-6160, a novel, potent, and selective molecular glue degrader of VAV1. The Company plans to file an Investigational New Drug (IND) application for MRT-6160 in the first half of 2024 and to develop the molecule as a potential treatment for autoimmune diseases.
“MRT-6160 is a potent, orally bioavailable MGD designed to degrade VAV1, an important protein involved in the signaling pathways of T and B cells. Our in vitro studies have shown that MRT-6160 selectively degrades VAV1 without detectable effects on other proteins. By targeting VAV1, MRT-6160 attenuates multiple aspects of T- and B-cell function and inhibits disease progression in established in vivo models of autoimmunity,” said Owen Wallace, Ph.D., Chief Scientific Officer of Monte Rosa. “The underlying biology and our preclinical data both demonstrate that MRT-6160 acts as an immune modulator, which has the potential to avoid the broad immune suppression seen with other approaches. We look forward to progressing our clinical plan developed with the goal of providing early insights into safety, PK and PD, and proof of concept regarding differentiated effects on key immunomodulatory signaling pathways.”
“Our goal centers on pioneering therapeutically meaningful new drugs for patients with serious diseases. By addressing VAV1, a validated but previously undruggable target, we believe we've created a potentially groundbreaking therapy for patients suffering from a range of serious autoimmune conditions, particularly those involving both T- and B cell-mediated autoimmunity,” said Markus Warmuth, M.D., CEO of Monte Rosa. “MRT-6160 is expected to be our second MGD to enter clinical trials, showcasing the continued productivity of our QuEEN™ platform. We anticipate significant progress and milestones in our portfolio in the upcoming year, including initial clinical data from our GSPT1 MGD, MRT-2359, in the second half of this year and filing of an IND application for MRT-6160 in the first half of next year.”
About Monte Rosa
Monte Rosa Therapeutics is a biotechnology company developing novel molecular glue degrader (MGD) medicines for patients with serious diseases such as oncology, autoimmune and inflammatory diseases. MGDs are small molecule protein degraders that employ the body’s natural mechanisms to selectively eliminate therapeutically relevant proteins. The Company’s QuEEN™ (Quantitative and Engineered Elimination of Neosubstrates) platform enables it to rapidly identify protein targets and design highly selective degraders by combining diverse libraries of proprietary MGDs with in-house proteomics, structural biology, AI/machine learning, and computational chemistry capabilities.
CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Globenewswire | May 02, 2023
Editas Medicine, Inc. a clinical stage genome editing company, announced that the journal Nature Biotechnology published the comprehensive data from a study of the proprietary SLEEK gene editing technology.
Despite major progress in achieving gene disruption with CRISPR-Cas gene editing technologies, efficient knock-in of transgenes continues to be a significant challenge for the gene editing field. To solve this challenge, SLEEK was developed to enable high knock-in efficiency with both viral and non-viral transgene formats while also ensuring robust simultaneous expression of up to four transgene cargos.
The study demonstrated that utilizing SLEEK results in the knock-in of multiple clinically relevant transgenes through a proprietary process that specifically selects only those cells containing the knock-in cargo. This process was developed by leveraging Editas Medicine’s proprietary engineered AsCas12a nuclease, which can achieve very high editing efficiency while maintaining high specificity. More than 90 percent knock-in efficiencies were observed in various clinically relevant target cells, including T cells, B cells, iPSCs, and NK cells. Additionally, SLEEK can be used to fine-tune the expression levels of transgene cargos, an important feature of next-generation cell therapies. As a demonstration of SLEEK’s potential value in clinical applications, the study authors used SLEEK to generate iPSC-derived NK cells capable of high-levels of in vivo persistence and robust tumor clearance in a solid tumor animal model.
“We are thrilled Nature Biotechnology published our paper sharing the comprehensive data on our SLEEK gene editing technology as we believe SLEEK has immense potential for gene editing drug development. As shared in the publication, SLEEK technology enables nearly 100 percent knock-in of functional transgene cargos at specific locations in the genome which may result in highly efficient multi-transgene knock-in for the next generation of cell therapy medicines,” said John A. Zuris, Ph.D., Director of Editing Technologies, Editas Medicine, and senior author on the study.
Editas Medicine believes that SLEEK may enable better product purity as well as shorter manufacturing timelines for the next generation of cell therapy medicines. Earlier this year, the Company announced it licensed its interest in the SLEEK technology to Shoreline Biosciences for specific usage in iPSC-derived NK and iPSC-derived macrophage cell therapies for oncology. The SLEEK technology remains an important Editas capability in iPSC engineering for a wide variety of future applications.
About Editas Medicine
As a clinical stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard University’s Cas9 patent estates and Broad Institute’s Cas12a patent estate for human medicines.
INDUSTRIAL IMPACT, MEDICAL
prnewswire | April 28, 2023
Hansa Biopharma AB, "Hansa" a pioneer in enzyme technology for rare immunological conditions, and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, announced they have entered a research and development collaboration.
The collaboration will, in a clinical study, evaluate the safety and efficacy of Hansa's antibody cleaving enzyme imlifidase as a pre-treatment prior to the administration of Genethon's gene therapy product candidate GNT-0003 in Crigler-Najjar syndrome in patients with pre-existing neutralizing antibodies (NAbs) to adeno-associated virus serotype 8 (AAV8). The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.
Søren Tulstrup, President and CEO, Hansa Biopharma stated: "Genethon is a pioneer at the cutting-edge of research and development of gene therapies for rare diseases and we are thrilled to be collaborating with them. This research collaboration further validates Hansa's commitment in gene therapy and underscores the important role that our antibody-cleaving enzyme technology can play in ensuring that even more patients can benefit from life-saving gene therapies".
GNT-0003 is currently being evaluated in a pivotal clinical study in France, Italy, and the Netherlands and has received PRIME (PRIority MEdicines) status from the EMA. Through the collaboration announced today, patients with Crigler-Najjar and pre-formed antibodies to AAV8 will be enrolled in a study with similar design where imlifidase is evaluated as a pre-treatment to enable gene therapy treatment with GNT-0003. The outcome of the ongoing clinical study of GNT-0003 could potentially form the basis for a MAA or BLA application in Europe or the US.
Crigler-Najjar is a genetic disease-causing bilirubin accumulation which leads to irreversible neurological damage manifested as muscle weakness, lethargy, deafness, mental retardation, and eye movement paralysis. Crigler-Najjar syndrome is an ultra-rare disease affecting less than one case per one million people per year.1
Frédéric Revah, CEO, Genethon added: "Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy. This collaboration with Hansa Biopharma is thus an important next step in the development of our gene therapy treatment for Crigler-Najjar syndrome, Hansa Biopharma's proven enzyme technology coupled with its scientific expertise will help us advance the critical research we are conducting in Crigler-Najjar and could enable gene therapy treatment for patients who are today not eligible because of their immunological status".
About Hansa Biopharma
Hansa Biopharma is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. Hansa has developed a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients. Hansa has a rich and expanding research and development program based on the Company's proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in transplantation, autoimmune diseases, gene therapy and cancer. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The Company is listed on Nasdaq Stockholm under the ticker HNSA.