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Details:
The collaboration aims to accelerate the development of optimized experimental antisense oligonucleotide (ASO) medicines for pediatric patients with extremely rare genetic neurological disorders.
Lead Product(s): Antisense Oligonucleotide
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Large molecule
Recipient: St. Jude Children’s Research Hospital
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration October 04, 2023
Details:
The funding will support n-Lorem’s efforts to discover optimal ASO candidates for several nano-rare patients. Once identified, n-Lorem will conduct preclinical studies necessary to support clinical development for each optimized ASO.
Lead Product(s): Antisense Oligonucleotide
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Large molecule
Partner/Sponsor/Collaborator: The Conrad Prebys Foundation
Deal Size: $0.3 million Upfront Cash: Undisclosed
Deal Type: Funding March 21, 2022
Details:
Partnership will support the n-Lorem’s effort to discover and provide experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life.
Lead Product(s): Antisense Oligonucleotide
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Large molecule
Recipient: Parexel Biotech
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership February 23, 2022
Details:
The collaboration aim to discover and provide experimental antisense oligonucleotide (ASO) medicines to nano-rare disease patients (1 to 30 patients worldwide) for free, for life.
Lead Product(s): Antisense Oligonucleotide
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Large molecule
Recipient: Cytiva
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership February 08, 2022
Details:
The partnership will supports and reduces the cost of manufacturing of n-Lorem’s experimental antisense oligonucleotide (ASO) medicines developed for nano-rare disease patients (1 to 30 patients worldwide) for free, for life.
Lead Product(s): Antisense Oligonucleotides
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: UndisclosedProduct Type: Large molecule
Recipient: Argonaut Manufacturing Services
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership February 02, 2022
Details:
n-Lorem Foundation is the first and only foundation with a mission to provide potentially life-saving treatments to patients who have diseases caused by extremely rare mutations for free by coupling advanced genomic diagnostics with antisense oligonucleotides drugs.
Lead Product(s): Antisense oligonucleotide
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Large molecule
Recipient: Ultragenyx Pharmaceutical
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 05, 2021
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