Editforce, Inc.

An increase in research and development (R&D) activities in myotonic dystrophy is expected to drive market growth by enhancing the understanding of the disease, accelerating drug discovery, and creating innovative treatments. With more R&D, new therapeutic options such as gene therapies, antisense oligonucleotides, and targeted small molecules can be developed, addressing the unmet needs in myotonic dystrophy care. Increased R&D also attracts funding and partnerships, fostering a competitive environment that can lead to improved patient outcomes and market expansion. LAS VEGAS, Nov. 27, 2024 /PRNewswire/ -- DelveInsight's ' Myotonic Dystrophy Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline myotonic dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the myotonic dystrophy pipeline domain. Key Takeaways from the Myotonic Dystrophy Pipeline Report DelveInsight's myotonic dystrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for myotonic dystrophy treatment. Key myotonic dystrophy companies such as Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others are evaluating new myotonic dystrophy drugs to improve the treatment landscape. Promising myotonic dystrophy pipeline therapies such as AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others are under different phases of myotonic dystrophy clinical trials. On November 7, 2024, ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, announced that the US Food and Drug Administration has granted Rare Pediatric Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1. In November 2024, Dyne Therapeutics announced that the US Food and Drug Administration has cleared the Investigational New Drug application for DYNE-101, which is being evaluated in the ongoing, global Phase I/II ACHIEVE trial in adults with myotonic dystrophy type 1 (DM1). The ACHIEVE trial currently includes 56 participants and is fully enrolled through the 6.8 mg/kg Q8W cohort (approximate ASO dose). In August 2024, PepGen announced that both Health Canada and the United Kingdom Medicines and Healthcare products Regulatory Agency have cleared the Company's clinical trial application (CTA) submissions for the FREEDOM2 trial, and PepGen expects to initiate patient dosing in the second half of 2024. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled, MAD clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in Canada, the United Kingdom, and in the United States, subject to regulatory clearance for the treatment of myotonic dystrophy. In May 2024, Avidity Biosciences announced that the US Food and Drug Administration granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1. In April 2024, PepGen presented a poster on the PGN-EDODM1 program at the 14th International Myotonic Dystrophy Consortium (IDMC-14) Meeting titled "FREEDOM-DM1: Phase 1 Study Design to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1." In March 2024, Entrada achieved a milestone under its global collaboration with Vertex related to the clinical advancement of Vertex's Phase I/II clinical trial of VX-670, which triggered a USD 75 million payment. The Company expects to receive this payment in the second quarter of 2024. The Company is eligible to receive up to USD 485 million, inclusive of milestones achieved to date, for the successful achievement of certain research, development, regulatory and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement. In February 2024, PepGen Inc. announced that the US Food and Drug Administration had granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1. In January 2024, Juvena Therapeutics receives the FDA Orphan Drug Designation for JUV-161 for the treatment of Myotonic Dystrophy Type 1. Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report The myotonic dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage myotonic dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the myotonic dystrophy clinical trial landscape. Myotonic Dystrophy Overview Myotonic dystrophy is a genetic disorder characterized by progressive muscle wasting and weakness. It is caused by mutations in specific genes that lead to an abnormal expansion of repetitive DNA sequences. The two main types of myotonic dystrophy are Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2). DM1 is caused by a CTG repeat expansion in the DMPK gene, while DM2 is linked to a CCTG repeat expansion in the CNBP gene. Symptoms of myotonic dystrophy can vary but often include muscle stiffness (myotonia), weakness, and atrophy, primarily affecting the distal muscles and those of the face. Additional symptoms may involve cataracts, cardiac arrhythmias, endocrine issues, and cognitive difficulties. Diagnosis typically involves a combination of clinical evaluation, family history assessment, and genetic testing to identify the specific mutations. Electromyography can also be used to detect myotonia. Currently, there is no cure for myotonic dystrophy, and treatment focuses on managing symptoms. This may include physical therapy to maintain muscle function, medications to alleviate myotonia, and regular monitoring for cardiac issues. Genetic counseling is recommended for affected individuals and their families to understand the inheritance patterns and potential risks. Find out more about myotonic dystrophy treatment drugs @ Drugs for Myotonic Dystrophy Treatment A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report: Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials Myotonic Dystrophy Therapeutics Assessment The myotonic dystrophy pipeline report proffers an integral view of the myotonic dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Myotonic Dystrophy Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Myotonin protein kinase expression inhibitors, RNA interference, Sodium channel antagonists, Glycogen synthase kinase 3 beta inhibitors, Histamine H3 receptor antagonists, Histamine H3 receptor inverse agonists, MicroRNA inhibitor, RNA inhibitors, RNA splicing modulators Key Myotonic Dystrophy Companies: Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others. Key Myotonic Dystrophy Pipeline Therapies: AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others. Dive deep into rich insights for new drugs for myotonic dystrophy treatment, visit @ Myotonic Dystrophy Drugs Table of Contents For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs Related Reports Myotonic Dystrophy Market Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key myotonic dystrophy companies, including Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, among others. Myotonic Dystrophy Epidemiology Forecast Myotonic Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted myotonic dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Duchenne muscular dystrophy companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics , among others. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

FUKUOKA, Japan, Oct. 18, 2022 /PRNewswire/ -- EditForce, Inc. (hereinafter "EditForce"), headquartered in Fukuoka, Japan, has realized the world's first RNA-editing technology that enables RNA-editing bases to be changed from "U" to "C," and demonstrated that this technology works even in human cells in a joint study with Professor Takahiro Nakamura, Faculty of Agriculture, Kyushu University. The study opens up the possibility of editing gene mutations which could not have been the target with the existing technologies, and is expected to contribute to the R&D of therapies for various genetic diseases. EditForce will improve safety and editing efficiency in its development activities to establish innovative gene therapy technologies. The study was published in the scientific journal "Communications Biology" on September 15 (September 16 in JST), 2022. Logo: Outline of research A genome existing in each human cell consists of four nucleotide bases -- A, C, G, and T -- of which the sequence holds the information necessary to make up a human body. Cells generate RNA consisting of four nucleotide bases -- A, C, G, and U -- based on genomic sequences, and then produce protein according to the sequences of RNA. However, changes in a single nucleotide on a genome or RNA sequence cause a variety of diseases. Treatment of these diseases requires single base-editing technology of repairing genomic or RNA mutations to bring the sequences back on the normal track. Currently, genome-editing technology has been developed rapidly, but the development of editing technology for RNA sequences remains limited. Especially as regards a single base substitution, technologies have been established to substitute "C" with "U" and "A" with "G," but the substitution of other bases is yet to be realized. The study clarified the mechanism of RNA editing in plants to substitute "U" with "C," based on which EditForce has realized the world's first RNA-editing (base substitution) technology that substitutes "U" with "C," and the company demonstrated that this technology works in human cells. Base-editing technologies can be applied to the treatment of diseases caused by a single mutation, and "U-to-C" RNA-editing technology of the study opens up the possibility to edit mutations which could not have been the target with the existing technologies. Further, repairing mutations by RNA editing without changes in genome sequence will enable EditForce to provide safer treatments to patients. The company expects that this technology will establish a novel gene therapy by improving safety and editing efficiency in its development activities. This work was supported by the Research Grant for Young Scholars funded by Yamanashi Prefecture. Article information Ichinose M., Kawabata M., Akaiwa Y., Shimajiri Y., Nakamura I., Tamai T., Nakamura T., Yagi Y. and Gutmann B. "U-to-C RNA editing by synthetic PPR-DYW proteins in bacteria and human culture cells" Commun Biol. 5, 968 (2022). About EditForce, Inc. EditForce, Inc., a Kyushu University-originated venture company developing a unique DNA/RNA-editing technology (PPR platform technology (*)), was established in May 2015 by KISCO Ltd. and Prof. Takahiro Nakamura of Kyushu University (former president of EditForce and currently its scientific advisor) and funded by companies and funds that have track records of investing in life sciences and biotechnology. EditForce aims for drug discovery that applies the PPR technology through joint research with universities and private companies. For more information, please visit: (*) Pentatricopeptide repeat (PPR) protein platform technology PPR is a protein discovered in plants that regulates gene expression by binding to DNA and RNA in a sequence-specific manner. The PPR proteins are also found in humans and yeasts, and they have similar functions. Prof. Takahiro Nakamura and Dr. Yusuke Yagi, CTO of EditForce, have focused on the PPR proteins and elucidated the mechanism that determines sequence specificity, and established a technology for creating various PPR proteins, each of which binds to a specific target DNA or RNA sequence. Furthermore, it is possible to manipulate and modify the target genome and RNA both inside and outside the cell by fusion with effector proteins. SOURCE EditForce, Inc.

After an immuno-oncology antibody deal , a cancer immunotherapy tie-up and a protein pact , Astellas Pharma is on to its next partnership, this time with a small UK biotech to treat eye diseases. The Japanese pharma will team up with Mogrify to find in vivo regenerative medicine approaches to treating sensorineural hearing loss. The goal is to find new combinations of transcription factors to help develop new cochlear hair cells. Astellas will foot the research costs and lend its AAV expertise through its gene therapy division, and Mogrify will provide its screening and validation process to help find potential therapies, the companies said Tuesday, without disclosing financial details. Mogrify picked up $17 million in additional financing last May. — Kyle LaHucik Mitsubishi Tanabe Pharma, which recently received an FDA green light for its oral ALS drug, will partner with a little-known Japanese biotech on potential gene therapies for an undisclosed central nervous system target. EditForce could receive more than 20 billion yen ($147 million) as part of the agreement, in exchange for giving the exclusive rights to Mitsubishi Tanabe for drug candidate molecules worldwide. Mitsubishi Tanabe will handle preclinical, clinical, manufacturing and commercialization work. The duo kept many details under wraps, including naming the specific CNS disease and timelines for clinical entry. The gene therapies will be developed using EditForce’s PPR protein platform, the companies said. — Kyle LaHucik After originally coming together in May 2018, Brii Biosciences likes what it sees in partner Vir Biotechnology and has decided to act upon its option to acquire Greater China rights to develop and market an HBV monoclonal antibody. The antibody, dubbed both VIR-3434 and BRII-877, is currently in Phase II development and is the second option exercise under the multi-year deal. Two more options remain under the infectious disease agreement. “Recently announced data by Vir showed that BRII-835 (VIR-2218) and BRII-877 (VIR-3434) are additive to each other resulting in a robust reduction of hepatitis B surface antigen (HBsAg), with no clinically significant safety signals for the combination,” Brii CMO Li Yan said in a statement. — Kyle LaHucik India-based biotech Aurigene has a new partner to work with. Aurigene is teaming up with EQRx to develop oncology and immune-inflammatory disease drugs, the biotechs announced in a statement Tuesday. Aurigene will lead discovery and preclinical efforts while EQRx, which is all about getting fast-follow drugs to the market at an affordable price, will take on clinical development, manufacturing and regulatory/commercialization efforts. Financial details of the agreement were not disclosed. “This agreement with EQRx further validates Aurigene’s proven expertise in discovery and preclinical development of novel therapeutics and moves Aurigene forward into building a global oncology franchise participating in the end-to-end value chain,” Aurigene CEO Murali Ramachandra said in a statement. A subsidiary of drug manufacturing giant Dr. Reddy’s Laboratory, Aurigene is based in Bengaluru and Kuala Lumpur. The company says it has contributed to 16 different drugs currently in clinical trials and previously partnered with Exelixis back in 2019. — Max Gelman