Polpharma European CDMO Partner & API Manufacturer since 1951 Polpharma European CDMO Partner & API Manufacturer since 1951

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million","upfrontCash":"$5.0 million","newsHeadline":"2seventy bio and Novo Nordisk Collaboration Delivers Key Proof of Concept Data, Triggering $15 Million Preclinical Milestone in In Vivo Gene Editing Hemophilia A Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"2Seventy Bio"}]

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            Details:

            The collaboration is focused on an in vivo gene editing treatment for hemophilia A by utilizing 2seventy bio’s megaTAL technology that has the potential to provide a highly specific and efficient way to silence, edit, or insert genetic components.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Novo Nordisk

            Deal Size: $40.0 million Upfront Cash: $5.0 million

            Deal Type: Collaboration May 01, 2023

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            The Collaboration to combine Korro’s RNA editing technology with Genevant’s industry-leading LNP technology to develop a differentiated therapeutic option for patients with Alpha-1 Antitrypsin Deficiency (AATD).

            Lead Product(s): RNA-based Therapeutic

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: Genevant Sciences

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration March 07, 2023

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            FT-002 is an intraocular injection of recombinant AAV virus carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function. It treat patients with X-linked retinitis pigmentosa caused by RPGR gene mutation.

            Lead Product(s): FT-002

            Therapeutic Area: Genetic Disease Product Name: FT-002

            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 February 17, 2023

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            Under the terms of the agreement, Curamys obtains non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Curamys

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement December 05, 2022

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            Mytesi (crofelemer) is an antidiarrheal indicated in the U.S. for the symptomatic relief of noninfectious diarrhea in adult patients with HIV/AIDS on antiretroviral therapy (ART). Mytesi is not indicated for the treatment of infectious diarrhea.

            Lead Product(s): Crofelemer

            Therapeutic Area: Genetic Disease Product Name: Mytesi

            Highest Development Status: Undisclosed Product Type: Small molecule

            Partner/Sponsor/Collaborator: Napo Therapeutics

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 17, 2022

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            Exosomes are small membrane vesicles, which are among the most productive and conducive to wound healing and countering inflammation. The benefits will help treat the skin and address signs of aging when delivered as a booster through Hydrafacial’s exceptional delivery system.

            Lead Product(s): Exosome Based Therapeutic

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: Beauty Health

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration September 14, 2022

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            Using patient-derived retina organoids, the discovered that a type of LCA caused by mutations in the AAV-based NPHP5 Gene Thearapy (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body.

            Lead Product(s): AAV-based NPHP5 Gene Thearapy

            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 September 08, 2022

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            Replay’s synHSV™ technology, a high payload capacity HSV vector capable of delivering up to 30 times the payload of AAV, is utilized by Replay’s four gene therapy product companies, bringing big DNA treatments to diseases affecting the skin, eye, brain, and muscle.

            Lead Product(s): AAV-based Gene Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: KKR

            Deal Size: $55.0 million Upfront Cash: Undisclosed

            Deal Type: Financing July 25, 2022

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            Funding supports Virica’s collaboration with the National Research Council of Canada’s Cell and Gene Therapy Challenge program to enhance the manufacturing of an affordable, AAV-LPL, a gene therapy being developed for people with debilitating lipoprotein lipase deficiency.

            Lead Product(s): Re-engineered AAV-LPL

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Innovation, Science and Economic Development Canada

            Deal Size: $0.4 million Upfront Cash: Undisclosed

            Deal Type: Collaboration May 02, 2022

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            CYTOO pioneered MyoScreen™, an in vitro R&D platform that enables the evaluation of therapies in patient-derived primary skeletal muscle cells under physiological conditions.

            Lead Product(s): AAV-based Gene Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Astellas Gene Therapies

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 05, 2022

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