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Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Undisclosed
Partner/Sponsor/Collaborator: Eli Lilly
Deal Size: $555.0 million Upfront Cash: $100.0 million
Deal Type: Licensing Agreement November 20, 2020
Details:
Lilly will use Precision's proprietary ARCUS® genome editing platform for the research and development of potential in vivo therapies for genetic disorders, with an initial focus on Duchenne muscular dystrophy and two other undisclosed gene targets.
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Lead Product(s): Gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Sarepta Therapeutics
Deal Size: $107.0 million Upfront Cash: Undisclosed
Deal Type: Series A Financing October 22, 2020
Details:
AavantiBio will pursue a diversified gene therapy pipeline, including a lead program in Friedreich’s Ataxia (FA), a rare inherited genetic disease characterized by cardiac and central nervous system dysfunction.
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Lead Product(s): Short hairpin RNA,Gaboxadol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: University of Connecticut
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration July 23, 2020
Details:
Ovid to collaborate with renowned molecular geneticist and Angelman syndrome expert Stormy J. Chamberlain, Ph.D., to advance a short hairpin RNA (shRNA)-based therapeutic with the goal of addressing the underlying genetic cause of Angelman syndrome.
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Lead Product(s): Transcend-peptide
Therapeutic Area: Genetic Disease Product Name: xB3
Highest Development Status: Undisclosed Product Type: Peptide
Partner/Sponsor/Collaborator: CHIESI USA INC
Deal Size: $141.0 million Upfront Cash: $3.0 million
Deal Type: Licensing Agreement June 29, 2020
Details:
The unique delivery method of their xB3 platform has the potential to overcome a significant challenge in the treatment of many neurological disorders, which is the ability to cross the blood brain barrier.
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Lead Product(s): Gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Personalis
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration June 02, 2020
Details:
As part of this research collaboration, Sarepta will be working with the Personalis team to characterize immune response to precision genetic therapeutics, utilizing Personalis’ advanced proprietary analytics.
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Lead Product(s): Gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Undisclosed
Deal Size: $1.0 million Upfront Cash: Undisclosed
Deal Type: Funding June 02, 2020
Details:
PPMD and Duchenne UK invite proposals which are investigating ways to mitigate the immune response both in terms of pre-existing immunity and immunity induced by receiving a gene therapy dose.
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Lead Product(s): Stem cell gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 02, 2020
Details:
Initially, the alliance will develop treatment options for patients with two rare, life-threatening primary immunodeficiency diseases -- Wiskott-Aldrich Syndrome (WAS) and X-linked Agammaglobulinemia (XLA).
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Lead Product(s): rAAVrh8-HexA/HexB
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 07, 2020
Details:
Presentations to showcase Axovant's investigational gene therapy programs, AXO-Lenti-PD and AXO-AAV-GM2.
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Lead Product(s): AAV capsid-gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Vertex Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 27, 2020
Details:
Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.
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Lead Product(s): Non-viral gene therapy
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Undisclosed Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: CureDuchenne Ventures
Deal Size: $1.0 million Upfront Cash: Undisclosed
Deal Type: Financing April 22, 2020
Details:
Myosana will use this initial funding to advance development of its innovative non-viral gene therapy to deliver full length dystrophin for Duchenne muscular dystrophy (DMD).
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