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                            Glycomine

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                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : CTI Life Sciences Fund

                            Deal Size : $115.0 million

                            Deal Type : Series C Financing

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                            Glycomine Raises $115M to Advance GLM101 Into Phase 2b Trial for PMM2-CDG

                            Details : The proceeds will advance GLM101, a first-in-class mannose-1-phosphate replacement therapy, is in development for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG).

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            April 16, 2025

                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : CTI Life Sciences Fund

                            Deal Size : $115.0 million

                            Deal Type : Series C Financing

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                            02

                            Denali Therapeutics

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Denali Therapeutics

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Denali Starts BLA Filing for Tividenofusp Alfa in Hunter Syndrome Treatment

                            Details : DNL310 (tividenofusp alfa) is composed of the iduronate 2-sulfatase enzyme fused to Denali’s proprietary ETV, designed to deliver IDS, addressing symptoms of Hunter syndrome.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            April 02, 2025

                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Glycomine

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Glycomine

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                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 24, 2025

                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Regenxbio

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Regenxbio

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : RGX-202

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            REGENXBIO Reports Positive Biomarker Data from AFFINITY DUCHENNE® Trial

                            Details : RGX-202 is a cell and gene therapy drug that works by transferring the micro-dystrophin gene, It is infused intravenously for the treatment of Duchenne Muscular Dystrophy.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 19, 2025

                            Lead Product(s) : RGX-202

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            QED Therapeutics

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            QED Therapeutics

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : Infigratinib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            An Interventional Study of Infigratinib in Children With Hypochondroplasia

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 12, 2025

                            Lead Product(s) : Infigratinib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Skyhawk Therapeutics

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Skyhawk Therapeutics

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : SKY-0515

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 12, 2025

                            Lead Product(s) : SKY-0515

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            PTC Therapeutics

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            PTC Therapeutics

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                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : Vatiquinone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            PTC Announces FDA Acceptance and Priority Review for Vatiquinone NDA

                            Details : PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), which is being developed for the treatment of Friedreich ataxia.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 19, 2025

                            Lead Product(s) : Vatiquinone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Denali Therapeutics

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed

                            Denali Therapeutics

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Denali Reports Primary & Long-Term Data on Tividenofusp Alfa in MPS II

                            Details : DNL310 (tividenofusp alfa), a fusion protein composed of IDS fused to Denali’s proprietary Enzyme transport vehicle. It is being evaluated in the treatment of Mucopolysaccharidosis Type II.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 06, 2025

                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Disc medicine

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed

                            Disc medicine

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $225.5 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Disc Medicine Prices $225.5M Upsized Public Offering Of Common Stock

                            Details : The net proceeds from the offering to fund R&D product candidates, to support the potential commercialization of DISC-1459 (bitopertin) for erythropoietic protoporphyria & X-linked protoporphyria.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 22, 2025

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $225.5 million

                            Deal Type : Public Offering

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                            10

                            Disc medicine

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed

                            Disc medicine

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Disc Medicine Shares FDA Type C Meeting Success for Bitopertin in EPP, NDA Plans

                            Details : DISC-1459 (bitopertin) is an investigational, orall inhibitor of GlyT1 that is designed to modulate heme biosynthesis. It is being evaluated for the treatment of erythropoietic protoporphyria.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 21, 2025

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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