Quotient Sciences Quotient Sciences

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and Gene therapy","graph2":"Benitec Biopharma"},{"orgOrder":0,"company":"Novadip Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novadip Biosciences receives IND approval from the FDA for regenerative bone product NVD-003","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"BELGIUM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Novadip Biosciences"},{"orgOrder":0,"company":"InnoCare Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InnoCare Announces Approval of Phase II Clinical Trial Using Orelabrutinib for the Treatment of NMOSD in China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND 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Announces US FDA Approval for a Phase 1 Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"SOUTH KOREA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"ImmunAbs"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"World's First CAR-T for NMOSD Treatment, IASO Biotherapeutics' Equecabtagene Autoleucel, Receives IND Approval by NMPA","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IASO Bio"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","amount":"$18.0 million","upfrontCash":"Undisclosed","newsHeadline":"Benitec Biopharma Inc. 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            SGX945 (dusquetide) is an innate defense regulator (p62 protein modulator), a new class of short, synthetic peptides. It is being evaluated for the treatment of lesions of Behçet's Disease

            Lead Product(s): Dusquetide

            Therapeutic Area: Rare Diseases and Disorders Product Name: SGX945

            Highest Development Status: IND Enabling Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2024

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            SGX945 (dusquetide) is an innate defense regulator (IDR), a new class of short, synthetic peptides. It is being evaluated in IND enabling studies for the treatment of oral lesions of Behçet's Disease.

            Lead Product(s): Dusquetide

            Therapeutic Area: Rare Diseases and Disorders Product Name: SGX945

            Highest Development Status: IND Enabling Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2024

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            PAX-101 (suramin sodium) is a intravenous formulation for Autism Spectrum Disorder. Suramin is a broadly acting anti-purinergic therapy and has reported positive results from a dose range study.

            Lead Product(s): Suramin

            Therapeutic Area: Rare Diseases and Disorders Product Name: PAX-101

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 27, 2023

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            The funding will be used to advance the development of Noregen (CTR-107), a novel regenerative therapeutic for the treatment of retinal-related vision loss due to a wide range of inherited and age-related diseases.

            Lead Product(s): CTR-107

            Therapeutic Area: Rare Diseases and Disorders Product Name: Noregen

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: National Institutes of Health

            Deal Size: $1.4 million Upfront Cash: Undisclosed

            Deal Type: Funding July 17, 2023

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            BB-301 is a novel, modified AAV9 capsid expressing bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 and two small inhibitory RNAs against mutant PABPN1 is currently in development for Oculopharyngeal Muscular Dystrophy.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            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 June 26, 2023

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            The collaboration will evaluate DISC-1459 (bitopertin), an orally administered inhibitor of GlyT1 that is designed to modulate heme biosynthesis, in a phase 2 clinical study of patients with Diamond-Blackfan anemia (DBA).

            Lead Product(s): Bitopertin

            Therapeutic Area: Rare Diseases and Disorders Product Name: DISC-1459

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: National Institutes of Health

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration March 21, 2023

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            The Company intends to use the net proceeds from this financing for the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB301 treatment study.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

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

            Partner/Sponsor/Collaborator: JMP Securities

            Deal Size: $18.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering September 16, 2022

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            The company intends to use the proceeds for development of BB-301, including the natural history lead-in study and Phase 1b/2a BB-301 treatment study, for continued advancement of development activities, for general corporate purposes and for strategic growth opportunities.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

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

            Partner/Sponsor/Collaborator: JMP Securities

            Deal Size: $18.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering September 12, 2022

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            Equecabtagene Autoleucel (CT103A) is a BCMA chimeric antigen receptor autologous T cell injection, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3ζ activation domains.

            Lead Product(s): Equecabtagene Autoleucel

            Therapeutic Area: Rare Diseases and Disorders Product Name: CT103A

            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 August 19, 2022

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            IM-101, a monoclonal antibody targeting complements that play essential roles in the immune system but, when out of regulation, cause various autoimmune diseases, including but not limited to paroxysmal nocturnal hemoglobinuria (PNH).

            Lead Product(s): IM-101

            Therapeutic Area: Rare Diseases and Disorders Product Name: IM-101

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 03, 2022

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