Modalis has been selected as a finalist in the XPRIZE Healthspan FSHD Bonus Prize Competition and awarded research funds.
Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® epigenome editing technology, announced its recognition as a top eight finalist in the prestigious XPRIZE Healthspan competition’s FSHD Bonus Prize and award of $250,000 for demonstrating a feasible solution to treat Facioscapulohumeral muscular dystrophy (FSHD) .
JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy
JCR Pharmaceuticals Co., Ltd. (TOKYO:4552; “JCR”) and Modalis Therapeutics Corporation (TOKYO:4883; “Modalis”) today announced that they have validated the initial proof of concept in a joint research program for the development of a novel gene therapy for a central nervous system (CNS) disease. Due to the success of the partnership thus far, Modalis and JCR have agreed to proceed to the next phase of their research by entering into a new joint research agreement.
Modalis Therapeutics has received an Orphan Drug Designation from the U.S. Food and Drug Administration for MDL-101, a treatment for congenital muscular dystrophy type 1A (LAMA2-CMD).
Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883, CEO: Haru Morita), a leading company pioneering treatments for rare genetic diseases using its proprietary CRISPR-based epigenome editing technology, CRISPR-GNDM®, today announced that the U.S. Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) to MDL-101, a novel precision medicine being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD).
Modalis Therapeutics: FDA Grants Rare Pediatric Disease Designation to MDL-101 for the Treatment of Congenital Muscular Dystrophy Type 1a (LAMA2-CMD)
Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883, CEO: Haru Morita), a leading company pioneering treatments for rare genetic diseases using its proprietary CRISPR-based epigenome editing technology, CRISPR-GNDM®, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to MDL-101, a novel precision medicine being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD).
Modalis Therapeutics Reports Data Supporting Development of a Transformative Epigenome Editing Therapeutic, MDL-101: a First-in-Class Epigenome Editing approach for the Treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2-CMD)
Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative drugs for the treatment of rare genetic diseases, utilizing its proprietary CRISPR-based epigenome editing technology CRISPR-GNDM®, posted the preprint paper titled “Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo” on bioRxiv. The company reported data demonstrating exceptional durability, robust efficacy and safety in dyW mouse disease model of LAMA2-CMD and in adult and juvenile NHPs.
Modalis Announces Partnership with Ginkgo Bioworks
Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) announced that it has entered into an agreement with Ginkgo Bioworks (Ginkgo) to join the Ginkgo Technology Network. This strategic alliance will enable Ginkgo’s global customers in the cell and gene therapy fields to access Modalis’ proprietary epigenome modulation technology, CRISPR-GNDM® (Guide Nucleotide-Directed Modulation). Modalis’ CRISPR-GNDM® Technology is capable of activating or suppressing any gene in a controlled manner without requiring DNA cleavage. This collaboration also creates a significant opportunity for Modalis to establish additional strategic partnerships.
Modalis and JCR Pharmaceuticals enter into joint research agreement
Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) today announced that it has entered into a joint research agreement with JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) on joint evaluation of the drug delivery technology of the gene therapy to the central nervous system (CNS) assuming CNS diseases as a target. Taking this agreement as an opportunity, Modalis and JCR Pharmaceuticals aim to establish the new gene therapies for undisclosed CNS diseases by applying J-Brain Cargo®, JCR Pharmaceuticals’ proprietary technology for crossing the blood-brain barrier (BBB), and CRISPR-GNDM® (Guide Nucleotide-Directed Modulation), Modalis proprietary epigenome modulation technology which does not require DNA cleavage.
Modalis Therapeutics to Present Data Supporting of Development of Transformative Epigenetic Editing Medicines for the Treatment of a Type of Muscular Dystrophy at the ASGCT Annual Meeting
Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® epigenetic editing technology, today announced that the abstract has been accepted for a presentation in the late-breaking session at the 26th Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT), being held in Los Angeles CA on May 16-20, 2023. The abstracts present preclinical data from the Company’s LAMA2 Congenital Muscular Dystrophy (LAMA2-CMD).
Modalis Therapeutics to Present Data Supporting of Development of Transformative Epigenetic Modulating Medicines for the Treatment of a Type of Muscular Dystrophy and the Other Genetic Disorders at the ASGCT Annual Meeting
Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® epigenetic modulating technology, today announced that six scientific abstracts have been accepted for presentation at the 25th Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT), being held in Washington D.C. and virtually, May 16-19, 2022. The abstracts present preclinical data from the Company’s Congenital Muscular Dystrophy type 1a (CMD1A) and the other rare disease programs in cardiovascular and neuroscience indications, as well as validation of our technology.
