Table Of Content
DT-216 muscle exposure in FA patients was lower than projected from animal studies but was sufficient to result in significant PD response in skeletal muscle. Initial data from the Phase 1 MAD trial is based upon a data cutoff of August 7, 2023. The study is fully enrolled but currently ongoing, with blinded PK and pharmacodynamic (PD) data available in the full 100 and 200mg dose cohorts and 11 of 14 participants in the 300mg dose cohort. Ms. Burgess brings more than 25 years of experience in financial reporting, technical accounting, debt and equity financings, and financial planning and management for public and private biotechnology companies.
Faculty
Prior to that, Dr. Jeffries established and led the informatics group at Kalypsys. Dr. Jeffries completed his Ph.D. at the University of Cambridge in epigenetics as an NIH-Cambridge scholar. Sarepta’s gene therapy engine provides a framework for creating a steady stream of new therapies for devastating diseases, such as limb-girdle muscular dystrophies.
Our Programs
Design is pioneering the field of small molecule genomic medicine, which holds the potential to safely and effectively deliver meaningful change to patients in need. Dr. William has served as Managing Partner and Chief Investment Officer of Logos Capital since its founding in 2019. Prior to founding Logos, Dr. Williams served as an investment professional at Farallon Capital Management.
Small Molecule
Design Therapeutics, Inc. (NASDAQ:DSGN) Given Average Recommendation of "Hold" by Analysts - MarketBeat
Design Therapeutics, Inc. (NASDAQ:DSGN) Given Average Recommendation of "Hold" by Analysts.
Posted: Sun, 14 Apr 2024 08:12:25 GMT [source]
Discovery efforts for multiple other small molecule genomic medicines are also underway. We believe our experiences with GeneTAC™ molecules allow us to more rapidly design GeneTAC™ molecules for additional indications. Our DM1 GeneTAC™ small molecules were shown to reduce observable CUG nuclear foci and correct splicing defects in DM1 patient-derived myotubes. We are working toward selection of a development candidate in anticipation of a future IND submission. Fuchs Endothelial Corneal Dystrophy (FECD) is a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells (CECs) and progressive loss of vision. Typically, the disease manifests after age 40 and can be detected through routine eye exams.
The platform is also being built to transform every laboratory and clinical experiment into a vehicle for rich data generation. The product development process itself fuels the development of improved AI models and biological foundation models. The structures of GeneTAC™ molecules enable them to bind and act specifically at the site of the disease-causing nucleotide repeat expansion. They target the mutant gene and modulate the cell’s native transcriptional machinery, selectively halting or resuming the expression of specific genes and in turn blocking pathogenic mRNA or protein production and restoring functional protein production. FA is a multisystem degenerative disease caused by a GAA nucleotide repeat expansion in the FXN gene that impairs transcription and reduces FXN mRNA.
Nisa Investment Advisors LLC increased its stake in shares of Design Therapeutics by 38,433.3% in the fourth quarter. Nisa Investment Advisors LLC now owns 11,560 shares of the company's stock worth $31,000 after purchasing an additional 11,530 shares in the last quarter. SG Americas Securities LLC purchased a new stake in shares of Design Therapeutics in the third quarter worth $47,000. Corton Capital Inc. increased its stake in shares of Design Therapeutics by 56.6% in the third quarter.
Deepa Prasad serves as an independent advisor to Équilibre Biopharmaceuticals Corp. and Graviton Biosciences. Prior to vTv, Ms. Prasad was managing director at WestRiver Group, where she led investments in healthcare innovation across biotech and digital health/AI. Ms. Prasad has held various healthcare operational roles as chief of staff at Blue Shield of California, regional vice president for Optum, head of managed care at the California Hospital Association, and vice president, financial strategy and business development at Coherus Biosciences (CHRS). She began her career in investment banking working with biotech and pharma companies on private placements and buyside and sellside mergers and acquisitions, including cross-border M&A with several Japanese pharma.
She currently serves on the grant funding committee for UC Davis and as a charter member for TiE, a non-profit global network of entrepreneurs and venture capitalists. Ms. Prasad earned her bachelor’s degree in business administration at the University of California, Berkeley and her M.B.A. from the Kellogg School of Management at Northwestern University with emphasis in finance and health industry management. Xaira’s advances in biological machine learning are underpinned by the company’s ability to generate, integrate, and learn from vast multidimensional data sets that comprehensively characterize disease-relevant biology at all scales, from molecules to people.
These foci inhibit the ability of MBNL1 to process pre-mRNAs, which when mis-spliced disrupt muscle development and function that is characteristic of DM1. Design Therapeutics, Inc a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. As a result, the DM1 GeneTAC™ molecules are designed to prevent the formation of the CUG hairpin structures that trap splicing proteins and produce nuclear foci. As with our other programs, the DM1 program is designed to address the underlying cause of the disease and benefit from the favorable development advantages of small molecules. Pratik Shah, Ph.D., is co-founder, president, chief executive officer and chairperson of Design Therapeutics.
Currently, there are no approved disease modifying therapeutic options that address the cause of nucleotide repeat expansion diseases. Through our proprietary GeneTAC™ approach, we can design and develop therapeutic candidates that dial gene expression up or down without requiring gene editing. HD is caused by a mutation that leads to an increased number of CAG triplet repeats in Exon 1 of the Huntingtin (HTT) gene. Expression of mutant HTT (mtHTT) negatively affects many cellular functions, leading to neuronal death and brain atrophy as symptoms manifest. Wild-type HTT (wtHTT) is thought to be important for normal neuronal function in the adult central nervous system (CNS). It is reported to be involved in axonal transport, synaptic function and cell survival.
As an exploratory objective, the company also evaluated FXN expression including levels of FXN mRNA and protein in skeletal muscle biopsies obtained at pre-dose baseline and two and seven days after the third weekly dose. In the hands of an experienced team of drug developers, Xaira’s platform forms the basis of a robust therapeutic product development capability across multiple modalities. Its design capabilities, applied to previously difficult to drug targets, are the foundation for a pipeline of differentiated therapeutics.
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