Design Therapeutics Inc

Design Therapeutics Inc(DSGN)資訊與事件

$15.110

-0.139 (-0.92%)收盤時

DSGN 資訊

DSGN 事件

8/3 08:30

Design Therapeutics Reports Strong Operational Performance in Q2

"Design continued its strong operational execution in the second quarter building on the positive RESTORE-FA data reported in May. Those four-week data demonstrated the ability of DT-216P2 to increase endogenous frataxin and its potential to deliver a differentiated, best-in-disease therapy for Friedreich ataxia," said Pratik Shah, chairperson and chief executive officer of Design Therapeutics. "Today's updates reflect continued progress across our pipeline, from the evolution of the DT-216P2 clinical program to the recent initiation of DT-818 dosing in DM1 patients, demonstrating the momentum of our GeneTAC platform across multiple serious genetic diseases."

6/30 08:30

Design Therapeutics Initiates DT-818 Clinical Trial

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.

6/4 14:20

FDA Pledges to Prevent Political Interference in Meeting with Rare Disease Nonprofits

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).

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