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Rare Disease Deal Benchmarks

Gene therapy, SMA, Duchenne, hemophilia, and orphan drug deal benchmarks. Benchmarks derived from 112 verified transactions.

112
Total Deals
$676M
Avg Upfront
5
Deal Types
100%
Terms Disclosed

Deal Structure Distribution

55
license
36
acquisition
18
collaboration
2
option
1
co development

Recent Deals with Disclosed Terms

PartiesTypeUpfrontTotal ValueDate
Hanmi PharmEli Lilly and Company
sonefpeglutide
license$75M$1.2BJun 2026
OscotecAgios Pharmaceuticals
cevidoplenib
license$25MJun 2026
Hansa BiopharmaSERB Pharmaceuticals
Idefirix (imlifidase)
license$123M$129MMay 2026
BioCryst Pharmaceuticals, Inc.Neopharmed Gentili
navenibart
license$70M$345MMay 2026
SantheraNxera
Agamree
license$40M$205MApr 2026
RegenxbioNippon Shinyaku
rare disease gene therapy portfolio
license$110M$810MMar 2026
uniQureCSL
Hemgenix
license$450M$1.9BMar 2026
Amicus TherapeuticsBioMarin Pharmaceutical
Amicus Therapeutics
acquisition$4.8B$4.8BDec 2025
Inozyme PharmaBioMarin Pharmaceutical
Inozyme Pharma
acquisition$270M$270MJul 2025
Regulus TherapeuticsNovartis
microRNA therapeutics
acquisition$800M$800MJul 2025

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Get instant benchmarks for upfront payments, milestones, and royalties based on 112 verified rare disease transactions.

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