Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : D3 LLC
Deal Size : $1.4 million
Deal Type : Series A Financing
RevolKa Secures 210M JPY ($1.4M) Series A Extension to Advance Therapeutic R&D
Details : The funding will advance RevolKa’s drug discovery programs for rare diseases using its technology, aiProtein, a robust AI-driven protein engineering technology.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Undisclosed
May 19, 2025
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : D3 LLC
Deal Size : $1.4 million
Deal Type : Series A Financing
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Eli Lilly
Deal Size : $1,013.0 million
Deal Type : Collaboration
Creyon Bio and Lilly Enter into RNA-Targeted Oligo Therapy Development Collaboration
Details : Through the collaboration, Creyon will leverage its industry-first AI-Powered Oligo Engineering Engine to design and optimize new drug candidates for Lilly’s named targets.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : $13.0 million
April 29, 2025
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Eli Lilly
Deal Size : $1,013.0 million
Deal Type : Collaboration
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : AstraZeneca
Deal Size : $3,475.0 million
Deal Type : Collaboration
Syneron Bio Announces Strategic Collaboration with AstraZeneca
Details : Under this collaboration, AstraZeneca will gain access to Syneron's Synova platform to support the advancements of research programmes exploring possible future treatments of chronic disease.
Product Name : Undisclosed
Product Type : Peptide
Upfront Cash : $75.0 million
March 21, 2025
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : AstraZeneca
Deal Size : $3,475.0 million
Deal Type : Collaboration
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Sanofi
Deal Size : Undisclosed
Deal Type : Agreement
Healx Partners with Sanofi To Explore Rare Diseases with AI Drug Discovery
Details : Under the agreement, Sanofi will provide data related to a late stage discontinued asset to identify new disease indications using Healnet, Healx’s AI-driven drug discovery platform.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
November 13, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Sanofi
Deal Size : Undisclosed
Deal Type : Agreement
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Novo Nordisk
Deal Size : Undisclosed
Deal Type : Partnership
Novo Nordisk Bets $600M on NanoVation for Genetic Medicines Beyond Liver
Details : The partnership combines NanoVation’s proprietary lcLNP™ technology for RNA delivery with Novo Nordisk’s expertise in cardiometabolic and rare disease R&D and clinical translation.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : $600.0 million
September 18, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Novo Nordisk
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : UMass Chan Medical School
Deal Size : Undisclosed
Deal Type : Partnership
Andelyn Biosciences, UMass Partner on Cockayne Syndrome
Details : Through the partnership, Andelyn Biosciences will leverage suspension AAV Curator Platform to manufacture clinical grade AAV9-CSA vector to treat Cockayne syndrome.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
July 18, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : UMass Chan Medical School
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Rett Syndrome Research Trust
Deal Size : $1.0 million
Deal Type : Collaboration
ProQR and Rett Syndrome Research Trust Join Forces with Axiomer™ RNA Editing Collaboration
Details : The collaboration focuses on developing editing oligonucleotides (EONs) using ProQR’s Axiomer technology to target MECP2 and correct specific mutations.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
May 01, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Rett Syndrome Research Trust
Deal Size : $1.0 million
Deal Type : Collaboration
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : n-Lorem Foundation
Deal Size : Undisclosed
Deal Type : Partnership
n-Lorem Partners with Hongene for Personalized ASO Medicines
Details : The partnership will discover personalized antisense oligonucleotide medicines for nano-rare disease patients, with Hongene providing essential compounds to n-Lorem to offset development costs.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
April 24, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : n-Lorem Foundation
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Nippon Shinyaku
Deal Size : Undisclosed
Deal Type : Collaboration
MiNA Therapeutics Collaborates with Nippon Shinyaku On RNAa Therapies
Details : MiNA Therapeutics will use its RNAa algorithm and platform to identify RNAa molecules targeting rare CNS genetic diseases under the collaboration.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
April 04, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Nippon Shinyaku
Deal Size : Undisclosed
Deal Type : Collaboration
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : BioMarin Pharmaceutical
Deal Size : Undisclosed
Deal Type : Collaboration
CAMP4 Partners on Research to Develop RNA-Targeting Medicines for Rare Diseases
Details : The collaboration combines BioMarin’s expertise in drug development with CAMP4’s platform-based target discovery capabilities to advance novel regRNA-targeting medicines for rare genetic conditions.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
January 10, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : BioMarin Pharmaceutical
Deal Size : Undisclosed
Deal Type : Collaboration