Rare Disease Deal Benchmarks
Gene therapy, SMA, Duchenne, hemophilia, and orphan drug deal benchmarks. Benchmarks derived from 202 verified transactions.
202
Total Deals
$2.5B
Avg Upfront
5
Deal Types
100%
Terms Disclosed
Deal Structure Distribution
86
license
82
acquisition
31
collaboration
2
option
0
co development
Recent Deals with Disclosed Terms
| Parties | Type | Upfront | Total Value | Date |
|---|---|---|---|---|
Sangamo Therapeutics → Eli Lilly CNS gene therapies | license | $18M | $1.4B | Mar 2026 |
Regenxbio → Nippon Shinyaku rare disease gene therapy portfolio | license | $110M | $810M | Mar 2026 |
Sangamo → Pfizer hemophilia A program | license | $70M | $345M | Mar 2026 |
Amicus Therapeutics → BioMarin Amicus Therapeutics | acquisition | $4.8B | $4.8B | Mar 2026 |
uniQure → CSL Hemgenix | license | $450M | $1.9B | Mar 2026 |
Kezar Life Sciences → Enodia Therapeutics KZR-540 | license | $1M | $128M | Mar 2026 |
Amicus Therapeutics → BioMarin Galafold and Pombiliti+Opfolda | acquisition | $4.8B | $4.8B | Dec 2025 |
Amicus Therapeutics → BioMarin Galafold and Pombiliti + Opfolda | acquisition | $4.8B | $4.8B | Dec 2025 |
Amicus Therapeutics → BioMarin Amicus portfolio including Galafold and Pombiliti + Opfolda | acquisition | $4.8B | $4.8B | Dec 2025 |
Amicus Therapeutics → BioMarin rare enzyme deficiency portfolio | acquisition | $4.8B | $4.8B | Dec 2025 |
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