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Lead Product(s): Golimumab
Therapeutic Area: Neurology Product Name: ION373
Highest Development Status: Preclinical Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 30, 2020
Details:
ION373 is an Ionis-owned investigational antisense medicine designed to stop the mutated gene from producing excess GFAP. Earlier this year, the European Medicines Agency (EMA) granted orphan drug designation to ION373.
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Lead Product(s): M102
Therapeutic Area: Neurology Product Name: M102
Highest Development Status: Preclinical Product Type: Undisclosed
Partner/Sponsor/Collaborator: FightMND
Deal Size: $0.7 million Upfront Cash: Undisclosed
Deal Type: Funding September 29, 2020
Details:
The grant will support the translational development of M102, a drug candidate for the treatment of amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease.
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Lead Product(s): PBFT02
Therapeutic Area: Neurology Product Name: PBFT02
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 17, 2020
Details:
In the study, a single administration of an optimized adeno-associated virus containing the GRN gene resulted in elevated levels of progranulin in the brain and cerebral spinal fluid, normalized lysosomal enzyme expression and corrected microgliosis in a mouse models.
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Lead Product(s): RNA editing therapies
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Wu Capital
Deal Size: $91.5 million Upfront Cash: Undisclosed
Deal Type: Series A Financing September 10, 2020
Details:
This financing enables Korro to advance its lead program to IND filing and establish a broad portfolio of innovative RNA editing therapies. Korro’s OPERA platform, harnesses the body’s natural base editing system, specifically ADAR to make targeted edits to a single RNA base.
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Lead Product(s): 4-Chlorokynurenine,Probenecid
Therapeutic Area: Neurology Product Name: AV-101
Highest Development Status: Preclinical Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 03, 2020
Details:
The results of this new study complement previous preclinical data demonstrating the combination's potential to substantially increase the brain concentration of AV-101's active metabolite, 7-Cl-KYN, thereby reducing, rather than blocking, NMDA receptor signaling.
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Lead Product(s): KLS-13019
Therapeutic Area: Neurology Product Name: KLS-13019
Highest Development Status: Preclinical Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 25, 2020
Details:
With the PCT Patent, along with Kannalife’s novel lead drug candidate, KLS-13019, and its other novel therapeutic agents like Atopidine™, Kannalife now has near global coverage in the top pharmaceutical markets in the world.
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Lead Product(s): Dantrolene Sodium
Therapeutic Area: Neurology Product Name: Ryanodex
Highest Development Status: Preclinical Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 19, 2020
Details:
The article reported results from an academic-based study that demonstrated dantrolene sodium administered intranasally improved both memory and cognition in a mouse model of Alzheimer’s disease. Eagle markets a formulation of dantrolene sodium but did not sponsor the study.
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Lead Product(s): Tau and Alpha-synuclein vectorized antibody
Therapeutic Area: Neurology Product Name: Alpha-synuclein vectorized antibody
Highest Development Status: Preclinical Product Type: Large molecule
Partner/Sponsor/Collaborator: AbbVie Inc
Deal Size: $250.9 million Upfront Cash: Undisclosed
Deal Type: Termination August 03, 2020
Details:
Voyager retains full rights to the vectorization technology and certain novel vectorized antibodies developed as part of the collaborations.
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Lead Product(s): AAV based gene regulation therapy
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Novartis Pharmaceuticals Corporation
Deal Size: $795.0 million Upfront Cash: $75.0 million
Deal Type: Collaboration July 30, 2020
Details:
Sangamo’s propriety genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), aim to develop gene regulation therapies to address three neurodevelopmental targets, including autism spectrum disorder (ASD) and other neurodevelopmental disorders.
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Lead Product(s): Pterostilbene,Epigallocatechin Gallate,Sulforaphane
Therapeutic Area: Neurology Product Name: QuadraMune
Highest Development Status: Preclinical Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 28, 2020
Details:
Pterostilbene is one of four ingredients which comprise QuadraMune™, a nutraceutical being developed by the Company which is currently in a clinical trial for prevention of COVID-19.
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