Rare Disease Deal Benchmarks
Gene therapy, SMA, Duchenne, hemophilia, and orphan drug deal benchmarks. Benchmarks derived from 113 verified transactions.
113
Total Deals
$1.6B
Avg Upfront
5
Deal Types
100%
Terms Disclosed
Deal Structure Distribution
51
license
40
acquisition
19
collaboration
2
option
1
co development
Recent Deals with Disclosed Terms
| Parties | Type | Upfront | Total Value | Date |
|---|---|---|---|---|
Santhera → Nxera Agamree | license | $40M | $205M | Apr 2026 |
Regenxbio → Nippon Shinyaku rare disease gene therapy portfolio | license | $110M | $810M | Mar 2026 |
uniQure → CSL Hemgenix | license | $450M | $1.9B | Mar 2026 |
Amicus Therapeutics → BioMarin Pharmaceutical Amicus Therapeutics | acquisition | $4.8B | $4.8B | Dec 2025 |
Inozyme Pharma → BioMarin Pharmaceutical Inozyme Pharma | acquisition | $270M | $270M | Jul 2025 |
Regulus Therapeutics → Novartis microRNA therapeutics | acquisition | $800M | $800M | Jul 2025 |
Amicus → BioMarin Fabry programs | acquisition | $4.8B | $4.8B | Jul 2025 |
Sangamo → Eli Lilly STAC-BBB AAV capsid | license | $18M | $1.4B | Jul 2025 |
Arrowhead Pharmaceuticals → Sarepta Therapeutics clinical and preclinical programs | acquisition | $325M | $325M | Feb 2025 |
Amicus Therapeutics → BioMarin Galafold (migalastat) + pipeline | acquisition | $4.8B | $4.8B | Feb 2025 |
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