Altay Therapeutics LinkedIn · Nov 2025
Altay announced that the FDA granted Orphan Drug Designation to DX5057, described as the first and only oral small-molecule DUX4 inhibitor in development for FSHD.
Altay develops disease-modifying therapies targeting transcription…
Company research
Public company, workplace, funding, and market signals
Overview
Altay Therapeutics is a San Francisco-based biopharmaceutical company developing oral small-molecule inhibitors of transcription factors to address serious, underserved diseases. Its lead program is DX5057, an oral DUX4 inhibitor for FSHD, alongside a STAT3 program for inflammatory diseases and oncology.
Primary product
DX5057, an oral DUX4 inhibitor for facioscapulohumeral muscular dystrophy (FSHD)
Founded
2019
Headquarters
San Francisco, California, United States
Team size
1-10
Industry
Biotechnology Research
Sub-industry
Biopharmaceutical drug discovery
Offices
455 Mission Bay Blvd S, San Francisco, California 94158, United States (HQ)
San Francisco, California, United States
United States (3 offices)
Business model
drug discovery
drug development
Funding
Stage
Seed
Total raised
$3.4M
Latest round
Grant · Sep 2023
Latest amount
$275K
People & investors
Leadership
Executive Chair
Chief Executive Officer, Board Director
Founder and CSO
Founder
Board member
Board member
Chief Medical Officer
Toxicology
Regulatory
CMC/Process Chemist
Drug Product
DMPK
Project Manager
MedChem
Advisor
Work & culture
Product & market
Differentiators
- —Therapeutic-area-agnostic platform that finds novel allosteric sites on transcription factors
- —First oral small-molecule DUX4 inhibitor (DX5057)
- —Broad pipeline spanning FSHD, STAT3/inflammation, and oncology
- —Explicit focus on previously considered "undruggable" transcription factors
- —Uses genetics, structural work, AI, and chemoproteomics to identify druggable pockets
Technology
biochemical assays
next-generation computational biology
chemoproteomics
genetics
structural biology
AI
allosteric site discovery
small-molecule medicinal chemistry
Google Cloud
Google Cloud CDN
Wix
Customers
patients with FSHD
patients with inflammatory diseases
patients with cancer
Competitors
Excision BioTherapeutics
Cedilla Therapeutics
Scale & contact
Estimated revenue
Third-party estimates vary from roughly $0.75M to $10M annually; several sources place it in the low-single-digit millions.
Estimated monthly visits
1.3K
Latest news
Altay Therapeutics LinkedIn · May 2025
Altay said it was selected as one of eight finalists for the $10M FSHD Bonus Prize, with a $250,000 award to advance feasible FSHD solutions.
Ali Ozes LinkedIn · Nov 2024
Altay announced that Dr. Mike Grey joined as Executive Chairman of the Board.
PR Newswire · Dec 2023
Altay won the Astellas-sponsored Future Innovator Prize at MBC BioLabs, gaining access to lab space and Astellas resources.
GlobeNewswire · Sep 2022
SOLVE FSHD announced a US$3 million investment in Altay Therapeutics to accelerate development of a DUX4 inhibitor for FSHD.
Jobs and careers at Altay Therapeutics
0 jobs at Altay Therapeutics
No open positions
Check back later for new openings