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Prevail Therapeutics
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Precision exercised its option to regain rights for the programs, including its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits.


Lead Product(s): Undisclosed

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Recipient: Precision BioSciences

Deal Size: $555.0 million Upfront Cash: $100.0 million

Deal Type: Termination April 16, 2024

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Under the agreement, Prevail has been granted rights to evaluate certain proprietary adeno-associated virus (AAV) capsids developed by Sangamo and may exercise certain options to license these capsids for multiple undisclosed neurological targets.


Lead Product(s): Adeno-associated Virus Gene Therapy

Therapeutic Area: Neurology Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Recipient: Sangamo Therapeutics

Deal Size: $1,190.0 million Upfront Cash: Undisclosed

Deal Type: Agreement July 17, 2023

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Under the collaboration, Prevail gets exclusive rights to Scribe’s CRISPR X-Editing (XE) technologies for the development of in vivo therapies directed to specified genetic targets known to cause serious neurological and neuromuscular diseases.


Lead Product(s): CRISPR-based Genetic Medicine

Therapeutic Area: Neurology Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Recipient: Scribe Therapeutics

Deal Size: $1,575.0 million Upfront Cash: $75.0 million

Deal Type: Collaboration May 16, 2023

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Delivering AAV gene therapy systemically to target the CNS, while limiting exposure to non-target organs (such as the liver), has been a significant challenge in the gene therapy field.


Lead Product(s): AAV-based Gene Therapy

Therapeutic Area: Neurology Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Recipient: Capsida

Deal Size: $740.0 million Upfront Cash: $55.0 million

Deal Type: Collaboration January 04, 2023

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The collaboration will focus on the discovery and development of novel AAV capsids for CNS diseases. As a part of the collaboration, Prevail will receive rights to utilize one of Lacerta’s novel AAV capsids for select undisclosed CNS targets.


Lead Product(s): AAV Capsid-based Therapeutic

Therapeutic Area: Neurology Product Name: Undisclosed

Highest Development Status: DiscoveryProduct Type: Cell and Gene therapy

Recipient: Lacerta Therapeutics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration August 23, 2022

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Acquisition will set up a gene therapy program at Lilly, based on Prevail's portfolio of neuroscience assets, including the lead candidate, PR001, and will broaden Lilly's commitment to use novel modalities to address fatal genetic forms of neurodegenerative disease.


Lead Product(s): PR001,Methylprednisolone,Sirolimus

Therapeutic Area: Neurology Product Name: PR001

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Eli Lilly

Deal Size: $1,040.0 million Upfront Cash: Undisclosed

Deal Type: Acquisition January 22, 2021

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Acquisition will establish a gene therapy program at Lilly, anchored by Prevail's portfolio of neuroscience assets, and will broaden Lilly's commitment to use novel modalities to attempt to address otherwise fatal genetic forms of neurodegenerative disease.


Lead Product(s): PR001,Methylprednisolone,Sirolimus

Therapeutic Area: Neurology Product Name: PR001

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Eli Lilly

Deal Size: $1,040.0 million Upfront Cash: Undisclosed

Deal Type: Acquisition December 15, 2020

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PR006 is an investigational AAV9 gene therapy delivering the GRN gene and is being developed as a potential therapy for patients with frontotemporal dementia with GRN mutations.


Lead Product(s): PR006,Methylprednisolone,Sirolimus

Therapeutic Area: Neurology Product Name: PR006

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 30, 2020

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The Company expects to initiate enrollment of the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease patients in the fourth quarter of 2020 and currently anticipates it will provide the next update on PR001 biomarker and safety data for nGD in 2021.


Lead Product(s): PR001,Methylprednisolone,Sirolimus

Therapeutic Area: Neurology Product Name: PR001

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 18, 2020

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The Company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). Prevail expects to start enrollment of the PROVIDE Phase 1/2 clinical trial of PR001 in the second half of 2020.


Lead Product(s): PR001,Methylprednisolone,Sirolimus

Therapeutic Area: Neurology Product Name: PR001

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 27, 2020

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