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Design Therapeutics, Inc. (Nasdaq: DSGN) is a clinical-stage biopharmaceutical company focused on developing innovative GeneTAC™ molecules, a novel class of small-molecule gene-targeted chimera therapeutic candidates. These molecules are designed to modify diseases by addressing their underlying genetic causes, specifically targeting nucleotide repeat expansion mutations.
The company's lead product candidate, DT-216, is in development for treating Friedreich ataxia (FA), a multisystem degenerative disease caused by a GAA nucleotide repeat expansion in the FXN gene. Recent Phase 1 multiple-ascending dose (MAD) clinical trial results have shown promising outcomes, with DT-216 being generally well-tolerated and demonstrating a dose-related increase in frataxin (FXN) mRNA levels in skeletal muscle biopsies.
In addition to FA, Design Therapeutics is advancing its GeneTAC™ program for Fuchs endothelial corneal dystrophy (FECD) and other serious nucleotide repeat-driven monogenic diseases. The company is also conducting studies for conditions such as myotonic dystrophy type-1, fragile X syndrome, spinocerebellar ataxias, Huntington disease, spinobulbar muscular atrophy, and C9orf72-amyotrophic lateral sclerosis/frontotemporal dementia.
Design Therapeutics recently reported third quarter 2023 financial results and announced a strategic realignment to prioritize long-term growth, focusing capital resources on program spend and implementing cost savings. This strategic move is expected to extend the company's cash runway through at least the next five years.
The company is also preparing to submit an Investigational New Drug (IND) application for its novel DT-168 eye drop for patients with FECD. Design Therapeutics' innovative approach and solid financial position underscore its commitment to bringing transformative therapies to patients with serious degenerative genetic diseases.
For more information, visit designtx.com.
Design Therapeutics, Inc. (Nasdaq: DSGN) reported a net loss of $15 million and R&D expenses of $11.3 million for Q2 2022. With cash and securities totaling $359.4 million, the company maintains a solid financial position to support its multi-year operational plan. Upcoming milestones include initial data from the Phase 1 trial of DT-216 for Friedreich ataxia expected in Q4 2022, while clinical development for the treatment of myotonic dystrophy type-1 is anticipated in 2023. The company also advances its research on Fuchs endothelial corneal dystrophy.
Design Therapeutics, a clinical-stage biotechnology company focused on serious genetic diseases, announced that CEO João Siffert and COO Sean Jeffries will participate in a fireside chat at the 2022 Wedbush PacGrow Virtual Healthcare Conference on August 10, 2022, at 10:20 a.m. ET. A live webcast will be accessible on the company's investor website, with an archive available for 30 days post-event.
Design's GeneTAC™ platform aims to develop therapies addressing genetic disorders like Friedreich ataxia and myotonic dystrophy type-1, targeting disease-causing genes.
Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology firm focused on serious degenerative genetic diseases, announced that CEO João Siffert will present at the 2022 Jefferies Healthcare Conference on June 8, 2022, at 8:30 a.m. ET in New York City. A live webcast will be available on their investor website, archived for 30 days post-presentation. The company is known for its GeneTAC™ technology aimed at targeting genes responsible for diseases like Friedreich ataxia and myotonic dystrophy type-1.
Design Therapeutics, Inc. (Nasdaq: DSGN) reported significant advancements in its clinical and research pipeline, emphasizing the initiation of Phase 1 trials for its lead GeneTAC™ molecule, DT-216, aimed at treating Friedreich Ataxia. The company highlighted a robust cash position of $371.2 million, supporting its multi-year operational strategy. R&D expenses were $8.8 million for Q1 2022, contributing to a net loss of $13.3 million. DT-216 has been granted Fast Track designation by the FDA, expediting its development process, while promising results for Fuchs endothelial corneal dystrophy were also presented.
Design Therapeutics (Nasdaq: DSGN) has completed dosing in the Phase 1 clinical trial of DT-216 for Friedreich ataxia (FA). This novel GeneTAC™ small molecule targets the genetic mutation causing FA, aiming to restore frataxin gene expression. The FDA has granted DT-216 Fast Track designation, facilitating its development due to the urgent need for effective FA treatments. The randomized, double-blind study will assess safety and tolerability, with topline data expected in the second half of 2022. The Fast Track status allows for expedited communication with the FDA during drug development.
Design Therapeutics (Nasdaq: DSGN) reported its fourth quarter and full year 2021 results, highlighting a significant milestone with the FDA clearance of its IND for DT-216, a GeneTAC™ targeting Friedreich ataxia. Total cash and investments stand at $384.1 million, ensuring ample resources for upcoming trials and projects. Research and development expenses for Q4 were $7.3 million, with a net loss of $11.1 million. The company is poised to present promising preclinical data on GeneTAC™ for Fuchs endothelial corneal dystrophy at ARVO 2022.
Design Therapeutics has announced promising preclinical data for its GeneTAC™ small molecules targeting Fuchs endothelial corneal dystrophy (FECD). The data showed a reduction of up to 99% in toxic nuclear foci and restoration of normal splicing in affected corneal endothelial cells. FECD affects over one million people in the U.S., primarily due to a mutation in the TCF4 gene. Currently, no disease-modifying therapies exist, with corneal transplantation as the only treatment. The findings will be presented at the ARVO 2022 Annual Meeting, indicating a significant advancement in potential treatments for genetic disorders.
Design Therapeutics, Inc. (Nasdaq: DSGN) announced the FDA's clearance of its Investigational New Drug Application for DT-216, aimed at treating Friedreich ataxia. The Phase 1 clinical trial will evaluate the safety and tolerability of DT-216, with enrollment expected to begin shortly. This milestone underscores the potential of Design's GeneTAC™ platform for developing therapies addressing genetic disorders. DT-216 targets the GAA repeat expansion mutation to restore frataxin expression, vital for combating FA symptoms. Topline data from the trial is anticipated in the second half of 2022.
Design Therapeutics, a biotechnology company focused on developing treatments for degenerative genetic disorders, announced that CEO João Siffert and COO Sean Jeffries will participate in a fireside chat at the SVB Leerink 11th Annual Global Healthcare Conference on February 17, 2022, at 11:20 a.m. ET. The event will be available via live webcast on the company's investor site, with an archive accessible for 30 days post-event. Design Therapeutics utilizes its GeneTAC™ platform to treat conditions like Friedreich ataxia and myotonic dystrophy type-1.
Design Therapeutics, Inc. (Nasdaq: DSGN) has appointed Dr. Jae Kim as Chief Medical Officer. With extensive experience in clinical strategy and drug development, Dr. Kim will lead the company’s transition to clinical trials, starting with a Phase 1 trial for Friedreich ataxia in early 2022. Dr. Kim previously served at Avidity Biosciences and Alnylam Pharmaceuticals, contributing to significant therapeutic approvals. His appointment is expected to enhance Design's capabilities in addressing genetic disorders through its GeneTAC™ technology.
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