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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 (Nasdaq: DSGN), a clinical-stage biotechnology company focused on developing treatments for serious degenerative genetic diseases, has announced its participation in two major investor conferences. The company will present at the 2024 Jefferies London Healthcare Conference on November 19, 2024, at 10:00 a.m. GMT in London, and participate in a fireside chat at the Piper Sandler 36th Annual Healthcare Conference on December 3, 2024, at 11:30 a.m. ET in New York. Live webcasts will be available on the company's website and archived for at least 30 days after the presentations.
Design Therapeutics (NASDAQ: DSGN) announced its Q3 2024 financial results and pipeline updates. The company initiated a Phase 1 trial for FECD with data expected in H1 2025, and plans to start a Phase 1 trial for Friedreich Ataxia in H1 2025. The company reported R&D expenses of $11.9M, G&A expenses of $4.4M, and a net loss of $13.0M for Q3 2024. With $254.1M in cash and securities, Design Therapeutics expects to fund operations into 2029, supporting up to four potential clinical proof-of-concept data sets. The company is also advancing preclinical programs in Huntington's Disease and Myotonic Dystrophy Type-1.
Design Therapeutics (Nasdaq: DSGN), a biotech company focused on developing treatments for serious degenerative genetic diseases, has announced its participation in the 2024 Cantor Global Healthcare Conference. The company's management will engage in a fireside chat on Thursday, September 19, 2024, at 12:45 p.m. ET in New York.
Investors and interested parties can access a live webcast of the fireside chat through a provided link and on the company's website at www.designtx.com in the investors section. The webcast will remain archived for at least 30 days after the presentation, allowing for later viewing.
This participation in a major healthcare conference highlights Design Therapeutics' commitment to engaging with the investment community and sharing updates on their progress in developing innovative treatments for genetic diseases.
Design Therapeutics (Nasdaq: DSGN) reported its Q2 2024 financial results and provided updates on its GeneTAC™ portfolio. Key highlights include:
1. Friedreich Ataxia (FA) program DT-216P2 on track for patient trials in 2025.
2. Fuchs Endothelial Corneal Dystrophy (FECD) candidate DT-168 advancing to Phase 1 in 2024.
3. Ongoing research in Myotonic Dystrophy Type-1 (DM1) and Huntington's Disease (HD).
4. Q2 2024 financial results: R&D expenses $10.5M, G&A expenses $4.5M, net loss $11.8M.
5. Strong cash position of $261.0M, expected to fund operations into 2029.
The company aims to generate clinical proof-of-concept data across its portfolio, addressing serious degenerative genetic diseases with novel small molecule therapies.
Design Therapeutics announced their first quarter 2024 financial results and upcoming program milestones. They are advancing a new drug product for Friedreich Ataxia and Fuchs Endothelial Corneal Dystrophy towards clinical trials. The company is progressing pipeline programs for Huntington’s Disease and Myotonic Dystrophy Type-1. Design Therapeutics also highlighted their cash and securities of $270.7 million, supporting multi-year operating runway.
Design Therapeutics, a biotechnology company focused on treating degenerative genetic diseases, will be participating in the 2024 RBC Capital Markets Global Healthcare Conference. The company's management will be involved in a fireside chat scheduled for May 15, 2024, at 11:30 a.m. ET in New York. The event will be live webcasted and archived for 30 days.
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