Design Secures $125 Million to Advance Treatments for Repeat Expansion Disorders

Rare Daily Staff

Design Therapeutics said it completed a $125 million series B financing to advance its pipeline of genomic medicines to treat serious degenerative disorders caused by inherited nucleotide repeat expansions.

Nucleotide repeat disorders are a set of genetic disorders caused by repeat expansion in certain genes. Depending on where it is located, the repeat may cause a change in the regulation of gene expression or produce a toxic gene product, all of which are recognized as significant contributors to degenerative diseases.

Proceeds from the financing will be used to advance Design Therapeutics’ novel GeneTAC platform, licensed from the Wisconsin Alumni Research Foundation, which leverages the disease modifying potential of genomic medicines with proprietary advances in transcriptional regulation chemistry, to create a pipeline of GeneTAC small molecules. The company’s initial focus is on developing medicines for the treatment Friedreich ataxia, which is expected to enter clinical development in the first half of 2022, and myotonic dystrophy type-1.

Friedreich’s ataxia, the most common form of hereditary ataxia in the United States, affects about 1 in every 50,000 people. Patients with Friedreich’s ataxia have an expanded GAA repeat in the first intron of the FXN gene, which blocks transcription and limits production of the frataxin protein and leads to progressive and life-altering movement problems and shortened life expectancy. Design’s experimental therapeutic unblocks transcription, thereby restoring the natural production and function of frataxin.

Logos Capital led the financing with participation by existing investors Cormorant Asset Management, SR One, Quan Capital, and WestRiver Group. They were joined by new investors, including funds and accounts managed by BlackRock, Janus Henderson Investors, RA Capital Management, Surveyor Capital (a Citadel company), funds and accounts advised by T. Rowe Price Associates, Wellington Management, Avoro Capital Advisors, Vivo Capital, and also including a U.S.-based, healthcare-focused fund and a leading global investment firm.

“This capital raise provides important resources to efficiently advance our pipeline, including our lead programs in Friedreich ataxia and myotonic dystrophy type-1,” said João Siffert, president and CEO of Design Therapeutics.

In addition, Arsani William, managing partner and chief investment officer of Logos Capital, and John Schmid, industry veteran and former chief financial officer of Auspex Pharmaceuticals, have joined the company’s board of directors.

Photo: João Siffert, president and CEO of Design Therapeutics