FSHD Draws $172 Million in a Week: A Pill and an RNA Drug

Jeff's Daily Pick. A muscular dystrophy with no approved treatment just attracted $172 million in one week. One startup is betting on a daily pill, the other on RNA delivered to muscle.

In the United States, two companies developing treatments for facioscapulohumeral muscular dystrophy, or FSHD, announced financings in the same week of October 2026. FSHD weakens the muscles of the face, shoulders and upper arms, and there is no approved disease modifying therapy. Both programs target the root problem: a gene called DUX4 that is switched on in muscle when it should be silent.

Altay: a pill against DUX4

Altay Therapeutics of San Carlos, California, closed a $37 million Series A on October 14, co led by Sound Bioventures and HealthCap, with Fountain Healthcare Partners, Biogen New Ventures, Pappas Capital and Global BioAccess Fund. The money will take DX5057, an oral small molecule designed to bind DUX4 and block its function, through Phase 1 and a Phase 2 proof of concept trial. A Phase 1 safety study is expected to start in the first quarter of 2027. In a transgenic mouse model expressing human DUX4, the drug reduced DUX4 associated activity and markers of muscle injury and preserved muscle function, the company said. DX5057 has FDA orphan drug designation.

Soufflé: RNA delivered to muscle

Soufflé Therapeutics, based in the Boston area and co founded by Robert Langer, closed an oversubscribed $135 million Series B on October 13, led by Juvia Group with new investors including TCG Crossover, BB Biotech, Fidelity, Balyasny and a sovereign wealth fund, alongside existing backers such as Amgen Ventures, ARCH Venture Partners and Temasek. Its REMEDY platform delivers oligonucleotide drugs to specific cells outside the liver. The most advanced program targets striated muscle in FSHD; a second targets the phospholamban gene in heart muscle for heart failure with reduced ejection fraction; a third is undisclosed. The company raised a $200 million Series A in October 2025.

The two bets differ in a way that matters to patients. A daily pill reaches every muscle through the bloodstream, which suits a disease that affects many muscle groups; a targeted oligonucleotide may hit DUX4 harder but depends on reaching muscle cells efficiently. Neither has shown it can change the disease in people yet. The first readout on the calendar is Altay's Phase 1, starting in early 2027.