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            Details:

            Ilofotase alfa is a proprietary recombinant alkaline phosphatase, constructed from two human isoforms of alkaline phosphatase, which is investigated for enzyme replacement therapy in adult hypophosphatasia (HPP) patients.

            Lead Product(s): Ilofotase Alfa

            Therapeutic Area: Genetic Disease Product Name: recAP

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 11, 2023

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            Ilofotase alfa is a proprietary recombinant alkaline phosphatase, constructed from two human isoforms of alkaline phosphatase, which is investigated for cardiac surgery-associated renal damage.

            Lead Product(s): Ilofotase Alfa

            Therapeutic Area: Genetic Disease Product Name: recAP

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 26, 2023

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            Details:

            AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology, which is investigated for Huntington’s Disease.

            Lead Product(s): rAAV5-miHTT

            Therapeutic Area: Genetic Disease Product Name: AMT-130

            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 June 21, 2023

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            VO659 is an antisense oligonucleotide (ASO) investigational therapy designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases including huntington’s disease (HD), spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3).

            Lead Product(s): VO659

            Therapeutic Area: Genetic Disease Product Name: VO659

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 03, 2023

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            Details:

            No SAE related to AMT-130 (rAAV5-miHTT) were reported in patients. Two SAE unrelated AMT-130 occurred: a deep-vein thrombosis in elbow in patient that resolved with anticoagulant and transient post-operative delirium second patient that was resolved through supportive care.

            Lead Product(s): rAAV5-miHTT

            Therapeutic Area: Genetic Disease Product Name: AMT-130

            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 June 23, 2022

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            Details:

            The ongoing Phase Ib/II trial of AMT-130 comprising a recombinant AAV5 vector carrying a DNA cassette encoding a microRNA that lowers Huntingtin protein in Huntington’s disease patients in Europe will enroll 15 patients.

            Lead Product(s): rAAV5-miHTT

            Therapeutic Area: Genetic Disease Product Name: AMT-130

            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 March 21, 2022

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            Details:

            AMT-130 (rAAV5-miHTT) appears to be generally well tolerated with no significant safety issues after surgical administration for one year of follow up in four patients for the treatment of Huntington’s disease.

            Lead Product(s): rAAV5-miHTT

            Therapeutic Area: Genetic Disease Product Name: AMT-130

            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 December 16, 2021

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            Details:

            The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States. To date, six patients have been treated with AMT-130, and four patients received the imitation surgery.

            Lead Product(s): rAAV5-miHTT

            Therapeutic Area: Genetic Disease Product Name: AMT-130

            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 May 27, 2021

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            Details:

            A Phase I/II trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntington’s disease randomized to treatment with AMT-130 or an imitation surgery across two dose cohorts.

            Lead Product(s): rAAV5-miHTT

            Therapeutic Area: Genetic Disease Product Name: AMT-130

            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 October 13, 2020

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            Details:

            The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States. One patient has been treated with AMT-130, and one patient received the imitation surgery.

            Lead Product(s): rAAV5-miHTT

            Therapeutic Area: Genetic Disease Product Name: AMT-130

            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 September 25, 2020

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