Lead Product(s) : RNA-based Therapy
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.
Brand Name : Undisclosed
Molecule Type : Large molecule
Upfront Cash : $600.0 million
September 18, 2024
Lead Product(s) : RNA-based Therapy
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.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
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) : ASO-based Therapy
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : n-Lorem Foundation
Deal Size : Undisclosed
Deal Type : Partnership
Details : The partnership aims to discover and provide personalized experimental antisense oligonucleotide medicines to nano-rare disease patients. Hongene will provide amidites, compounds essential to developing ASO medicines, to n-Lorem to offset the costs of de...
Brand Name : Undiclosed
Molecule Type : Large molecule
Upfront Cash : Undisclosed
April 24, 2024
Lead Product(s) : ASO-based Therapy
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : n-Lorem Foundation
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : RNAa-based Therapy
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Nippon Shinyaku
Deal Size : Undisclosed
Deal Type : Collaboration
Details : Under the collaboration, MiNA Therapeutics will utilize its proprietary RNAa algorithm and technology platform to identify and characterise RNAa molecules targeting rare genetic diseases of the central nervous system (CNS).
Brand Name : Undisclosed
Molecule Type : Large molecule
Upfront Cash : Undisclosed
April 04, 2024
Lead Product(s) : RNAa-based Therapy
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Nippon Shinyaku
Deal Size : Undisclosed
Deal Type : Collaboration
Lead Product(s) : RNA Editing Therapeutic
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : ARCH Venture Partners
Deal Size : $30.0 million
Deal Type : Financing
AIRNA Launches to Develop New Class of RNA Editing Therapeutics for Rare and Common Diseases
Details : The financing will advance company's pipeline of RNA editing therapeutics driven by its flexible RNA editing platform, RESTORE+TM. AIRNA is advancing development of a best-in-class product candidate to treat the inherited genetic disease alpha-1 antitryp...
Brand Name : Undisclosed
Molecule Type : Large molecule
Upfront Cash : Undisclosed
September 19, 2023
Lead Product(s) : RNA Editing Therapeutic
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : ARCH Venture Partners
Deal Size : $30.0 million
Deal Type : Financing
Lead Product(s) : RNA-editing Therapeutic
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Sirnaomics
Deal Size : Undisclosed
Deal Type : Partnership
Sirnaomics Establishes a Strategic Partnership with EDIRNA Inc., an RNA-Editing Therapeutic Company
Details : The partnership accelerates EDIRNA's proprietary Edit-to-Cure Therapeutics™ platform for RNA-Editing therapeutics, targeting diseases with high unmet clinical needs.
Brand Name : Undisclosed
Molecule Type : Large molecule
Upfront Cash : Undisclosed
July 05, 2023
Lead Product(s) : RNA-editing Therapeutic
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Sirnaomics
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : Antibody-based Therapy
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Rallybio
Deal Size : Undisclosed
Deal Type : Collaboration
Details : Under the terms of the agreement, AbCellera and Rallybio will co-develop up to five rare disease novel antibody-based therapeutic targets, which will be chosen together by both companies.
Brand Name : Undisclosed
Molecule Type : Large molecule
Upfront Cash : Undisclosed
December 01, 2022
Lead Product(s) : Antibody-based Therapy
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Rallybio
Deal Size : Undisclosed
Deal Type : Collaboration
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Mitobridge
Deal Size : $180.0 million
Deal Type : Collaboration
Details : Under the terms of the agreement, the companies will jointly conduct research and preclinical development activities to identify novel monovalent small molecules that modulate target proteins through activation, stabilization or degradation as potential ...
Brand Name : Undisclosed
Molecule Type : Small molecule
Upfront Cash : Undisclosed
June 29, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Mitobridge
Deal Size : $180.0 million
Deal Type : Collaboration
Lead Product(s) : AAV9-based Gene Therapy
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Adeno-Associated Virus Engineering Platform, Platform utilizes combinatorial engineering and automation to screen in high-throughput, generating capsids greatly improved over AAV9 in CNS efficiency and specificity.
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
May 18, 2022
Lead Product(s) : AAV9-based Gene Therapy
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : GSK
Deal Size : $1,150.0 million
Deal Type : Collaboration
Details : The collaboration will combine Adrestia’s world leading, synthetic viability platform with GSK’s deep scientific expertise in human genetics, functional genomics, screening, and bioinformatics.
Brand Name : Undisclosed
Molecule Type : Undisclosed
Upfront Cash : Undisclosed
December 18, 2020
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : GSK
Deal Size : $1,150.0 million
Deal Type : Collaboration
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