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    [{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Publishes Paper on Novel Therapeutic Approach for Treatment of All Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Wave Life Sciences to Present Preclinical In Vivo and In Vitro Data for SNP3-Targeting Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large 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Announces $200 Million Private Placement Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Tyra Biosciences"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Neurocrine Biosciences","pharmaFlowCategory":"D","amount":"$1,865.0 million","upfrontCash":"$165.0 million","newsHeadline":"Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich's Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Voyager Therapeutics, Inc"},{"orgOrder":0,"company":"Cyprium Therapeutics","sponsor":"National Institute of Neurological Disorders and Stroke","pharmaFlowCategory":"D","amount":"$4.1 million","upfrontCash":"Undisclosed","newsHeadline":"Fortress Biotech and Cyprium Therapeutics Announce $4.1 Million Grant from NINDS to Further Development of AAV-ATP7A Gene Therapy for Menkes Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Cyprium Therapeutics"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","amount":"$420.0 million","upfrontCash":"$100.0 million","newsHeadline":"Intellia Exits Hemophilia B Pact with Regeneron","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Genflow Biosciences","sponsor":"EXO Biologics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genflow Biosciences Partners with Revatis SA and EXO Biologics in Groundbreaking Aging Research, Fueled by Two Major Research Grants","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Genflow Biosciences"},{"orgOrder":0,"company":"Gain Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Gain Therapeutics Announces Preclinical Data Showing Restoration of Enzymatic Function with Novel Allosteric Regulators in GM1 Gangliosidosis Model","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Gain Therapeutics"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Be Bio Announces New Preclinical Data Demonstrating its Novel Engineered B Cell Medicine (BCM), BE-101, Produces Active and Sustained Levels of Factor IX for the Treatment of Hemophilia B\u200b","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Be Biopharma"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Be Biopharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"MaxCyte Signs Strategic Platform License with Be Biopharma to Support the Development of Engineered B Cell Medicines (BCMs)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"MaxCyte"},{"orgOrder":0,"company":"Diagonal Therapeutics","sponsor":"BVF Partners","pharmaFlowCategory":"D","amount":"$128.0 million","upfrontCash":"Undisclosed","newsHeadline":"Diagonal Therapeutics Launches with $128 Million in Financing to Pioneer a New Approach to Discovering and Developing Agonist Antibodies to Tackle the Underlying Causes of Severely Debilitating Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Diagonal Therapeutics"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Korro Announces Selection of its First Development Candidate for the Potential Treatment of Alpha-1 Antitrypsin Deficiency (AATD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Korro Bio"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Axovia Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Charles River & Axovia Partner to Support Development of Gene Therapies for Ciliopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Charles River Laboratories, Inc"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Prevail Therapeutics","pharmaFlowCategory":"D","amount":"$555.0 million","upfrontCash":"$100.0 million","newsHeadline":"Precision BioSciences Announces Return of Programs and Conclusion of Collaboration with Prevail Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Precision BioSciences"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Deep Track Capital","pharmaFlowCategory":"D","amount":"$70.0 million","upfrontCash":"Undisclosed","newsHeadline":"Korro Announces $70 Million Private Placement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Korro Bio"},{"orgOrder":0,"company":"hC Bioscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"hC Bioscience Announces Lead Program in Hemophilia and Reports Positive Preclinical Data on Novel Protein Editing Approach Using Anticodon Engineered tRNA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"hC Bioscience"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Metagenomi to Regain Full Development Rights to its Wholly-owned Base Editing and RIGS Systems","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Metagenomi"},{"orgOrder":0,"company":"Latus Bio","sponsor":"8VC","pharmaFlowCategory":"D","amount":"$54.0 million","upfrontCash":"Undisclosed","newsHeadline":"Latus Launches with State-of-the-Art Gene Therapy Development Technologies, Two Lead Product Candidates, and an Initial $54 Million in Series A Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Latus Bio"},{"orgOrder":0,"company":"Be Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Be Biopharma Announces New Preclinical Data for Novel B Cell Medicine for the Potential Treatment of Hypophosphatasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Be Biopharma"},{"orgOrder":0,"company":"Silence Therapeutics","sponsor":"Hansoh Pharma","pharmaFlowCategory":"D","amount":"$1,316.0 million","upfrontCash":"$16.0 million","newsHeadline":"Silence Therapeutics Achieves $2 Million Research Milestone Payment from Hansoh Pharma Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Silence Therapeutics"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","amount":"$1.7 million","upfrontCash":"Undisclosed","newsHeadline":"Opus Genetics Announces $1.7 Million in Funding for Preclinical Programs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Opus Genetics"},{"orgOrder":0,"company":"2Seventy Bio","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"2seventy bio Sells Hemophilia A Candidate and MegaTAL Technology to Novo Nordisk for $40M","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"2Seventy Bio"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Novel Pharma","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GC Biopharma\/Novel Pharma's Sanfilippo Syndrome Treatment Obtains FDA IND Approval","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SOUTH KOREA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"GC Biopharma"}]

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              Axplora

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              Sensorion and Novasep Announce Signature of a Gene Therapy Product Development and Manufacturing Agreement

              Details:

              Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

              Lead Product(s): OTOF-GT,Aav Vectors

              Therapeutic Area: Genetic Disease Product Name: OTOF-GT

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

              Partner/Sponsor/Collaborator: Sensorion

              Deal Size: Undisclosed Upfront Cash: Undisclosed

              Deal Type: Agreement October 27, 2020

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              2Seventy Bio

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              2seventy bio Sells Hemophilia A Candidate and MegaTAL Technology to Novo Nordisk for $40M

              Details:

              Novo has acquired the Hemophilia A program and rights to 2seventy’s in vivo gene editing technology for autologous or allogeneic cell therapies of immune cells for the treatment of autoimmune disease.

              Lead Product(s): Undisclosed

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

              Partner/Sponsor/Collaborator: Novo Nordisk

              Deal Size: $40.0 million Upfront Cash: Undisclosed

              Deal Type: Divestment June 26, 2024

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              Silence Therapeutics

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              Silence Therapeutics Achieves $2 Million Research Milestone Payment from Hansoh Pharma Collaboration

              Details:

              The collaboration aims to develop siRNAs leveraging Silence’s mRNAi GOLD platform for three undisclosed targets. Silence has exclusive rights to the first two targets in all territories except china.

              Lead Product(s): siRNA-based Therapy

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

              Highest Development Status: Preclinical Product Type: Large molecule

              Partner/Sponsor/Collaborator: Hansoh Pharma

              Deal Size: $1,316.0 million Upfront Cash: $16.0 million

              Deal Type: Collaboration June 24, 2024

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              Opus Genetics

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              Opus Genetics Announces $1.7 Million in Funding for Preclinical Programs

              Details:

              The proceeds will be used to conduct a preclinical safety study for OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations (RHO-adRP).

              Lead Product(s): OPGx-RHO

              Therapeutic Area: Genetic Disease Product Name: OPGx-RHO

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

              Partner/Sponsor/Collaborator: Foundation Fighting Blindness

              Deal Size: $1.7 million Upfront Cash: Undisclosed

              Deal Type: Funding June 13, 2024

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              GC Biopharma

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              GC Biopharma/Novel Pharma's Sanfilippo Syndrome Treatment Obtains FDA IND Approval

              Details:

              GC1130A (recombinant human heparan N-sulfatase), is an intracerebroventricular (ICV) enzyme replacement therapy candidate under investigation for the treatment of sanfilippo syndrome.

              Lead Product(s): Recombinant Human Heparan N-sulfatase

              Therapeutic Area: Genetic Disease Product Name: GC1130A

              Highest Development Status: Preclinical Product Type: Large molecule

              Partner/Sponsor/Collaborator: Novel Pharma

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable May 20, 2024

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              Latus Bio

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              Latus Launches with State-of-the-Art Gene Therapy Development Technologies, Two Lead Product Candidates, and an Initial $54 Million in Series A Financing

              Details:

              The financing aims to fund Latus Bio to focus on the research and development of their investigational product, which is being evaluated in the early-stage clinical trial studies for the treatment of type 2 neuronal ceroid lipofuscinosis.

              Lead Product(s): Undisclosed

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

              Partner/Sponsor/Collaborator: 8VC

              Deal Size: $54.0 million Upfront Cash: Undisclosed

              Deal Type: Series A Financing May 02, 2024

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              Metagenomi

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              Metagenomi to Regain Full Development Rights to its Wholly-owned Base Editing and RIGS Systems

              Details:

              Metagenomi and Moderna have mutually agreed to terminate their collaboration on primary hyperoxaluria type 1 (PH1), and rights to develop the PH1 program, as well as all other rights granted under the collaboration, will be returned as part of the termination.

              Lead Product(s): Undisclosed

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

              Partner/Sponsor/Collaborator: Moderna Therapeutics

              Deal Size: Undisclosed Upfront Cash: Undisclosed

              Deal Type: Termination May 01, 2024

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              hC Bioscience

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              hC Bioscience Announces Lead Program in Hemophilia and Reports Positive Preclinical Data on Novel Protein Editing Approach Using Anticodon Engineered tRNA

              Details:

              HCB-101 is an anticodon engineered tRNA designed to suppress nonsense mutations & is delivered as a lipid nanoparticle to target the liver,where Factor VIII is produced. It is being evaluated for the treatment of severe hemophilia A.

              Lead Product(s): HCB-101

              Therapeutic Area: Genetic Disease Product Name: HCB-101

              Highest Development Status: Preclinical Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable April 23, 2024

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              Korro Bio

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              Korro Announces $70 Million Private Placement

              Details:

              The net proceeds will be used to support the clinical advancement of Korro's lead product KRRO-110 for treating patients suffering from alpha-1 antitrypsin deficiency.

              Lead Product(s): KRRO-110

              Therapeutic Area: Genetic Disease Product Name: KRRO-110

              Highest Development Status: Preclinical Product Type: Large molecule

              Partner/Sponsor/Collaborator: Deep Track Capital

              Deal Size: $70.0 million Upfront Cash: Undisclosed

              Deal Type: Private Placement April 18, 2024

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              Charles River Laboratories, Inc

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              Charles River & Axovia Partner to Support Development of Gene Therapies for Ciliopathies

              Details:

              The collaboration aims to support the development of Axovia’s gene therapies, including AXV101, for ciliopathies, including Bardet-Biedl Syndrome (BBS), a condition with limited treatment options and no cure.

              Lead Product(s): AXV101

              Therapeutic Area: Genetic Disease Product Name: AXV101

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

              Partner/Sponsor/Collaborator: Axovia Therapeutics

              Deal Size: Undisclosed Upfront Cash: Undisclosed

              Deal Type: Collaboration April 09, 2024

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              Farmhispania Group, a leading European CDMO in HPAPI Technologies & High Potency Fermentation.
              One Pharma aims to be one of the leading generic companies in Europe.
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