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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) has announced promising preclinical results for its GeneTAC™ small molecule, DT-168, aimed at treating Fuchs endothelial corneal dystrophy (FECD). The eye drops reduced nuclear foci and improved mis-splicing in patient-derived corneal endothelial cells. Advanced FECD cases require surgeries like corneal transplants, and no disease-modifying therapies are currently available. DT-168 targets CTG repeat expansions in the TCF4 gene, marking its potential as a disease-modifying treatment. The company is set to submit an Investigational New Drug application for DT-168 in the second half of 2023, indicating a significant step in the therapeutic development process.
Design Therapeutics, Inc. (Nasdaq: DSGN) reported advancements across its pipeline in gene therapies during its fourth quarter and full-year 2022 earnings. The company is progressing in its Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich ataxia, with initial results expected mid-2023. Financially, Design Therapeutics maintains a strong position with $330.4 million in cash and securities, ensuring operations through 2025. R&D expenses were $48.6 million for 2022, contributing to a net loss of $63.3 million. Upcoming milestones include IND submissions for DT-168 and DM1 programs, aiming for clinical development over the next two years.
Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology firm focusing on treatments for degenerative genetic diseases, announced that CEO João Siffert will engage in a fireside chat during the SVB Securities Virtual Global Biopharma Conference on February 15, 2023, at 5:00 p.m. ET. This event will be accessible via a live webcast on the company’s website and will be archived for 30 days. Design Therapeutics is pioneering GeneTAC™ therapy aimed at modifying disease-causing gene expressions, currently testing its lead candidate DT-216 for Friedreich ataxia, alongside other programs targeting various genetic disorders.
Design Therapeutics (Nasdaq: DSGN) announced progress in its GeneTAC™ portfolio, including promising results for DT-216 in a Phase 1 trial for Friedreich ataxia (FA). The therapy demonstrated a >2-fold increase in frataxin mRNA, indicating effective gene transcription restoration. DT-216 was well-tolerated with no serious adverse events reported. The company also introduced DT-168 for treating Fuchs endothelial corneal dystrophy, with an IND submission expected in 2023, and aims for an IND for myotonic dystrophy type-1 in 2024.
Design Therapeutics (DSGN) reported significant progress in its clinical pipeline, particularly with DT-216 targeting Friedreich ataxia (FA). Initial data from the single-ascending dose trial is expected in December 2022, while the multiple-ascending dose trial has commenced, aiming for completion by mid-2023. The company, with a solid cash position of $344.2 million, continues to advance its GeneTAC™ platform across multiple diseases. Third-quarter financials show R&D expenses of $14.3 million and a net loss of $17.7 million.
Design Therapeutics is advancing its Friedreich ataxia (FA) treatment, DT-216, as it prepares to report initial Phase 1 trial data in Q4 2022. The company presented promising preclinical findings at the International Congress for Ataxia Research, indicating DT-216's potential to restore frataxin (FXN) gene expression and improve mitochondrial function. Significant results include a 10-fold increase in FXN mRNA in patient-derived cells and restored FXN protein levels in FA neurons. Design aims to share safety and pharmacokinetics data from the trial soon.
Design Therapeutics, Inc. (Nasdaq: DSGN) announced that its CEO, João Siffert, M.D., and COO, Sean Jeffries, Ph.D., will participate in a fireside chat at the 2022 Jefferies Cell and Genetic Medicine Summit on September 29, 2022, at 10:00 a.m. ET in NYC. A live webcast will be available on the investor section of the company's website and archived for 30 days. Design Therapeutics focuses on developing innovative therapies using its GeneTAC™ platform to treat degenerative genetic diseases, with lead programs targeting Friedreich ataxia and myotonic dystrophy type-1.
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