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                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Study Phase : Discovery

                          Sponsor : Global Blood Therapeutics

                          Deal Size : $353.0 million

                          Deal Type : Licensing Agreement

                          Details : These mechanisms of the two licensed novel small moleculeare are distinct and potentially complementary to that of Oxbryta tablets, a novel hemoglobin S polymerization inhibitor approved in the United States for the treatment of SCD in patients ages 12 y...

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                          March 16, 2021

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Global Blood Therapeutics

                          Deal Size : $353.0 million

                          Deal Type : Licensing Agreement

                          Sanofi Company Banner

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                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Under the collaboration, Sanofi has exercised its option for a second target as part of the companies' research collaboration to develop CRISPR-based therapeutics using Scribe’s CRISPR technologies.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : $40.0 million

                          January 13, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Sanofi

                          Deal Size : $1,240.0 million

                          Deal Type : Expanded Collaboration

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                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Through the collaboration, Vertex will utilize Orna's proprietary LNP delivery solutions to enhance it's efforts in developing next-gen gene editing therapies for patients with SCD and TDT.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : $65.0 million

                          January 07, 2025

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Vertex Pharmaceuticals

                          Deal Size : $700.0 million

                          Deal Type : Collaboration

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                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Under the collaboration, Sanofi has exercised its option for a second target as part of the companies' research collaboration to develop CRISPR-based therapeutics using Scribe’s CRISPR technologies.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : $40.0 million

                          March 01, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Sanofi

                          Deal Size : $1,240.0 million

                          Deal Type : Expanded Collaboration

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                          05

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Under the collaboration, GC4K will develop a gene therapy for the treatment of Hereditary Spastic Paraplegia Type 56 (SPG56), a progressive neurological disease characterised by varying degrees of spasticity and muscle weakness.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          November 28, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Genetic Cures for Kids

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          06

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Fulcrum gains worldwide license to intellectual property arising from CAMP4’s DBA program, including the rights for discovery, development, and commercialization of novel therapeutic agents against an undisclosed target for the potential treatment of D...

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          October 07, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Fulcrum Therapeutics

                          Deal Size : $70.0 million

                          Deal Type : Licensing Agreement

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                          07

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Gensaic will use the net proceeds to develop full CFTR gene delivery to lung tissue using its proprietary phage-derived particle (PDP) platform in CFTR-based gene Therapy.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          September 01, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Cystic Fibrosis Foundation

                          Deal Size : $3.5 million

                          Deal Type : Funding

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                          08

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : The collaboration aims to combine Genevant’s proprietary LNP technology with innovative mRNA-based megaTAL technology to develop an in vivo gene editing treatment for hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          June 11, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Novo Nordisk

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          09

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : As part of the research collaboration, the companies will leverage Pfizer's expertise and Gero’s technology platform with the aim to identify genes and pathways linked to fibrotic diseases.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          June 01, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Pfizer Inc

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          10

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Through the collaboration with Orbit, Sanegene is looking to develop a portfolio of advanced RNAi based Therapies. Orbit’s proprietary technology enables the screening of large libraries of peptides using a combination of DNA encoded libraries and bead...

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          May 01, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Discovery

                          Sponsor : Orbit Discovery

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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