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Human Genome Research Institute"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-Lenti-PD","moa":"Undisclosed","graph1":"Neurology","graph2":"Phase II","graph3":"Sio Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sio Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sio Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AXO-Lenti-PD","moa":"Undisclosed","graph1":"Neurology","graph2":"Phase II","graph3":"Sio Gene 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Find Clinical Drug Pipeline Developments & Deals by Sio Gene Therapies

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                          01

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1’s ability to increase β-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and exten...

                          Product Name : AXO-AAV-GM1

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          October 21, 2021

                          Lead Product(s) : AXO-AAV-GM1,Rituximab,Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : AXO-AAV-GM1, the company’s adeno-associated viral vector (AAV)9-based gene therapy for the treatment of Type I (early infantile onset) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.

                          Product Name : AXO-AAV-GM1

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 10, 2021

                          Lead Product(s) : AXO-AAV-GM1,Rituximab,Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Initial data from the ongoing Phase 1/2 study in five patients in the low-dose cohort showed that AXO-AAV-GM1 was generally well tolerated with a favorable safety profile and provide early indications of clinical disease stability.

                          Product Name : AXO-AAV-GM1

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 15, 2020

                          Lead Product(s) : AXO-AAV-GM1,Rituximab,Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : National Human Genome Research Institute

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : AXO-Lenti-PD gene therapy demonstrate consistent treatment outcomes including Favorable safety and tolerability profile, with no serious adverse events attributable to gene therapy; Improvement in Hauser diary “Good ON time” and “OFF time” and ot...

                          Product Name : OXB-102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          October 29, 2020

                          Lead Product(s) : AXO-Lenti-PD

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase II

                          Sponsor : Oxford Biomedica

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : AXO-AAV-GM2 is the first investigational gene therapy to achieve IND clearance for Tay-Sachs and Sandhoff diseases. The Company received a letter from the FDA indicating that it has satisfactorily addressed all issues related to the clinical hold.

                          Product Name : AXO-AAV-GM2

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 11, 2020

                          Lead Product(s) : AXO-AAV-GM2

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : AXO-AAV-GM1 is the first gene therapy to enter clinical trials for GM1 gangliosidosis and the company believes that it has the potential to provide meaningful clinical benefit to both Type I and Type II patients.

                          Product Name : AXO-AAV-GM1

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 10, 2020

                          Lead Product(s) : AXO-AAV-GM1

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Axovant has completed enrollment in the low-dose cohort of the Phase 1/2 study for Type II (late infantile and juvenile onset) GM1 patients evaluating safety, tolerability, and exploratory measures of efficacy at a dose of 1.5x1013 vg/kg delivered intrav...

                          Product Name : AXO-AAV-GM1

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 06, 2020

                          Lead Product(s) : AXO-AAV-GM1

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Presentations to showcase Axovant's investigational gene therapy programs, AXO-Lenti-PD and AXO-AAV-GM2.

                          Product Name : rAAVrh8-HexA/HexB

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          July 05, 2020

                          Lead Product(s) : rAAVrh8-HexA/HexB

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : A Phase 1/2 clinical trial testing AXO-Lenti-PD (OXB102), Axovant‘s experimental gene therapy for Parkinson’s disease, has completed dosing of all four adults in a second group in its dose-escalation part of the study.

                          Product Name : OXB-102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 15, 2020

                          Lead Product(s) : AXO-Lenti-PD

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : AXO-Lenti-PD was well-tolerated with no treatment-related serious adverse events at 6 months. EXPLORE-PD, a randomized, sham-controlled study of AXO-Lenti-PDis expected to begin dosing in 2021.

                          Product Name : OXB-102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 10, 2020

                          Lead Product(s) : AXO-Lenti-PD

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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