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[{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Therapeutics Announces Presentations at ASGCT and ARVO Annual Meetings","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Ophthalmology","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by U.S. FDA","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Ophthalmology","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Aavnergene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Neurophth and AAVnerGene Enter Strategic AAV Capsids Partnership for Next-Generation Ophthalmic Gene Therapy","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"Discovery Platform","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Ophthalmology","graph2":"Discovery Platform"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Announces IND Approval by the NMPA for Leber Hereditary Optic Neuropathy Gene Therapy","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Ophthalmology","graph2":"IND Enabling"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"CMG-SDIC Capital","pharmaFlowCategory":"D","amount":"$60.0 million","upfrontCash":"Undisclosed","newsHeadline":"Neurophth Raised Over $60 Million USD in Series-C Financing to Advance Gene Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Therapeutics Receives IND Clearance to Initiate Clinical Trial for China AAV-ND4 Gene Therapy NR082 in Leber Hereditary Optic Neuropathy","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Ophthalmology","graph2":"IND Enabling"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"China CDE Grants Breakthrough Therapy Designation (BTD) to Neurophth's NR082 in LHON","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Commercialization of Gene Therapy is coming soon: Neurophth Completes Patient Enrollment for Phase III clinical trial for the gene therapy treatment of LHON","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Yangtze River-CMB International Industry Fund","pharmaFlowCategory":"D","amount":"$95.0 million","upfrontCash":"Undisclosed","newsHeadline":"Neurophth Secures Nearly 95 Million USD in Series C+ Financing for Gene Therapy Clinical Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Announces First Patient Dosed in Phase I\/II Clinical Trial of Second Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Announces Completion of Patient Enrollment for Opvika\u00ae Phase I\/II Clinical Trial in the U.S.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"}]

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            Opvika (Esonadogene Imvoparvovec) is a MT-ND4 modulator. It is being evaluated for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON).

            Lead Product(s): Esonadogene Imvoparvovec

            Therapeutic Area: Genetic Disease Product Name: Opvika

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 19, 2024

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            NFS-02 is Neurophth’s second gene therapy, currently evaluating in multi-region, multi-center Phase I/II clinical trial for the treatment of Leber hereditary optic neuropathy caused by ND1 mutation.

            Lead Product(s): NFS-02

            Therapeutic Area: Genetic Disease Product Name: NFS-02

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 17, 2023

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            The net proceeds will advance clinical trials for Neurophth's core products, enhancing the firm's R&D capabilities and expanding its pipeline, including NFS-01 (esonadogene imvoparvovec), a rAAV2-ND4 gene therapy being developed to treat Leber's hereditary optic neuropathy.

            Lead Product(s): Esonadogene Imvoparvovec

            Therapeutic Area: Genetic Disease Product Name: NFS-01

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Yangtze River-CMB International Industry Fund

            Deal Size: $95.0 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing August 11, 2023

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            NR082 (rAAV2-ND4), a novel recombinant AAV2, containing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 gene which delivers the correct genes to the patients' damaged optic ganglion cells. It is being developed for LHON associated with mtND4 mutations.

            Lead Product(s): NR082

            Therapeutic Area: Genetic Disease Product Name: NR082

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 22, 2023

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            Investigational NFS-02, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 1 gene, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy.

            Lead Product(s): rAAV2-ND1

            Therapeutic Area: Genetic Disease Product Name: NFS-02

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 19, 2022

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            NR082 (rAAV2-ND4), a recombinant adeno-associated viral vector, serotype 2, containing human ND4 codon-optimized gene under the control of the cytomegalovirus promoter and enhancer, is a novel gene therapy.

            Lead Product(s): rAAV2-ND4

            Therapeutic Area: Genetic Disease Product Name: NR082

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 15, 2022

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            INDA Clearance received from USFDA for NFS-01 (NR082), a novel recombinant adeno-associated viral serotype 2 on the basis of results from three investigator-initiated trials which demonstrated favorable results in patients LHON associated ND4 mutation.

            Lead Product(s): NR082

            Therapeutic Area: Ophthalmology Product Name: NFS-01

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 18, 2022

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            Details:

            The financing will be used to progress its lead clinical program in LHON to ex-China, enrich R&D pipeline and programs through business development and continue to expand the international standard gene therapy manufacturing platform.

            Lead Product(s): rAAV-ND4

            Therapeutic Area: Genetic Disease Product Name: NFS-01

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: CMG-SDIC Capital

            Deal Size: $60.0 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing November 22, 2021

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            NR082, a novel recombinant adeno-associated viral serotype 2 containing a codon-optimized of ND4 gene under the control of the cytomegalovirus promoter and enhancer, is a novel ophthalmic injection that is being developed for the treatment of LHON in ND4 mutations.

            Lead Product(s): NR082

            Therapeutic Area: Ophthalmology Product Name: NFS-01

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 01, 2021

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            Details:

            Subject to the terms of the agreement, Neurophth will make an initial cash payment to AAVnerGene to test ~100 AAV capsids. Within 6-12 months upon receiving the AAV capsids, Neurophth is responsible for completing the preclinical studies on these capsids.

            Lead Product(s): AAV capsids-gene therapy

            Therapeutic Area: Ophthalmology Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Cell and Gene therapy

            Recipient: Aavnergene

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership January 19, 2021

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