Design Therapeutics Inc. has described transcription modulators reported to be useful for the treatment of myotonic dystrophy type 1 (DM1) and Fuchs’ dystrophy.
In the first five months of 2024, the BioWorld Neurological Diseases Index (BNDI) saw a 10.35% decline, underperforming both the Nasdaq Biotechnology Index (NBI), which rose by 0.7%, and the Dow Jones Industrial Average (DJIA), which increased by 2.64%. This represents an additional drop for BNDI, which closed February with a 4.1% decline. In contrast, in 2023, the BNDI closed with a 4.36% increase, outperforming the NBI, which rose by 3.74%, but not matching the 13.7% rise seen in the DJIA.
Research at Design Therapeutics Inc. has led to the discovery of conjugates consisting of a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence linked to a protein binding moiety through oligomeric backbone linker. They are transcription modulators reported to be useful for the treatment of Huntington’s disease.
Variable muscle expression of frataxin (FXN) protein in muscle plus five adverse events (AEs) of injection-site thrombophlebitis in the multiple ascending-dose (MAD) phase I study hamstrung Design Therapeutics Inc. in its Friedreich’s ataxia (FA) bid with DT-216. Wall Street punished the Carlsbad, Calif.-based firm’s stock (NASDAQ:DSGN) with a knockdown of 70%, or $5.17, leaving shares at $2.17 when the market closed Aug. 15.
A Design Therapeutics Inc. patent details conjugates consisting of DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence linked to protein binding moiety through oligomeric backbone linker.
The recent online publication of findings from the University of Southern California ataxia working group called Enigma served to fuel more interest in the simmering drug development space of Friederichs’s ataxia (FRDA), where a handful of gene therapies and other approaches, plus one promising small-molecule treatment, are in the works.
Three companies set terms for their IPOs that, if launched, will go far in maintaining the year’s already powerful momentum. Largest of the three comes from Design Therapeutics Inc., which looks to raise net proceeds of $209.1 million to fund its Friedreich’s ataxia program through IND studies and a phase I trial.
Design Therapeutics Inc., a San Diego startup developing new therapies for degenerative disorders caused by nucleotide repeat expansions, has raised $45 million in series A financing.
