DSGN News & Events

Design Therapeutics announced the initiation of patient dosing in its Phase 1 multiple-ascending dose clinical trial evaluating DT-818 in adults with myotonic dystrophy type-1. DT-818 is a GeneTAC small molecule designed to selectively reduce transcription of the mutant DMPK allele and address the underlying cause of DM1. The Phase 1 trial is an open-label, MAD study designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of DT-818 in adults with DM1. Design anticipates reporting data from this study in 2027.

Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff. In the meeting, Diamantas pledged to prevent political interference, Endpoint News' Zachary Brennan reports. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

The U.S. Food and Drug Administration said it has issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge. When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells. "Today's action reflects the FDA's commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options," said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research. "By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on.Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible." This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of complementary FDA actions in this area. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).