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Ovid Therapeutics

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Ovid and UConn Enter Strategic Research Collaboration

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.

Bioasis

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Bioasis and Chiesi Group Announce Rare Diseases Strategic Alliance

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.

Sarepta Therapeutics

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Personalis Announces Scientific Collaboration with Sarepta Therapeutics on Immune Response to Precision Genetic Therapeutics

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.

Duchenne research

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$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

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.

CSL Behring

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CSL Behring and Seattle Children's Research Institute to Advance Gene Therapy Treatments for Primary Immunodeficiency Diseases

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).

Axovant Gene Therapies

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Axovant Announces Upcoming Presentations at the Annual Meeting of the American Society of Gene and Cell Therapy

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.

Affinia Therapeutics

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Vertex Pharmaceuticals and Affinia Therapeutics Set Up Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies

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.

Myosana Therapeutics

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Myosana Therapeutics, Inc. Raises Up To $1M from CureDuchenne Ventures

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).

Arcturus Therapeutics

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Arcturus Therapeutics Announces Proposed Public Offering of Common Stock

Lead Product(s): mRNA

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Undisclosed Product Type: Large molecule

Partner/Sponsor/Collaborator: Guggenheim Securities

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Public Offering April 15, 2020

Details:

The Company intends to use the net proceeds for working capital including capital expenditures and research and development expenditures related to the advancement of LUNAR-OTC, LUNAR-COV19.

Dyne Therapeutics

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Dyne Therapeutics Announces Equity Investment from CureDuchenne Ventures

Lead Product(s): Oligonucleotide linked antibody

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Undisclosed Product Type: Large molecule

Partner/Sponsor/Collaborator: CureDuchenne Ventures

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Financing March 31, 2020

Details:

The funding will support Dyne’s development of precision therapeutics designed to restore muscle health in people living with Duchenne muscular dystrophy (DMD).

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Product Type

Companies

Ovid Therapeutics

Ovid and UConn Enter Strategic Research Collaboration

Bioasis

Bioasis and Chiesi Group Announce Rare Diseases Strategic Alliance

Sarepta Therapeutics

Personalis Announces Scientific Collaboration with Sarepta Therapeutics on Immune Response to Precision Genetic Therapeutics

Duchenne research

$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

CSL Behring

CSL Behring and Seattle Children's Research Institute to Advance Gene Therapy Treatments for Primary Immunodeficiency Diseases

Axovant Gene Therapies

Axovant Announces Upcoming Presentations at the Annual Meeting of the American Society of Gene and Cell Therapy

Affinia Therapeutics

Vertex Pharmaceuticals and Affinia Therapeutics Set Up Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies

Myosana Therapeutics

Myosana Therapeutics, Inc. Raises Up To $1M from CureDuchenne Ventures

Arcturus Therapeutics

Arcturus Therapeutics Announces Proposed Public Offering of Common Stock

Dyne Therapeutics

Dyne Therapeutics Announces Equity Investment from CureDuchenne Ventures