Eli Lilly and Company is both an existing shareholder and strategic partner. AX-0810 is advancing in cholestatic diseases with biliary atresia as the lead phase II indication, while next-generation programs like AX-0811 and AX-0422 show strong preclinical results.
Cyborg Score Rationale
The company has a GF Score of 44/100, indicating potential challenges in financial strength (3/10). However, analyst sentiment is positive with strong pipeline potential. Stock volatility and pre-commercial stage present execution risk.
Top Insights
In June 2026, ProQR raised ~$50M through registered direct offering with Eli Lilly acquiring 5.1M shares for ~$9.2M.
AX-0810 target engagement data in healthy volunteers was on track for Q2 2026; biliary atresia selected as initial Phase 2 indication.
The company focuses on advancing RNA therapeutics for severe genetic rare diseases; its Axiomer RNA base-editing platform and upcoming clinical data from first-in-human studies are expected to enhance valuation.
ProQR has 187 employees.
Named Competitors
Beam Therapeutics — Precision genetic medicine via base editing
CRISPR Therapeutics — Gene editing therapies using CRISPR-Cas9
Translate Bio — mRNA therapeutics for genetic diseases
Reata Pharmaceuticals — Small molecule therapies for rare genetic disorders
Recent Developments
(June 2026) $50M registered direct offering priced at $1.81 per share
(June 2026) Announced positive target engagement data from Phase 1 clinical study of AX-0810 oligonucleotide
(Q1 2026) Released Q1 2026 operating and financial results on May 12, 2026
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