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                            Sanofi

                            France
                            BIO International Convention
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

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                            Translate Bio Provides MRT5005 Program Updates

                            Details : Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.

                            Product Name : MRT5005

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis

                            Details : First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

                            Product Name : MRT5005

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            AccurEdit Therapeutics

                            China arrow
                            Peptide Therapy Summit
                            Not Confirmed

                            AccurEdit Therapeutics

                            China arrow
                            Peptide Therapy Summit
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : ART001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            AccurEdit’s ART001 Gene Editing Drug gets FDA Orphan Designation

                            Details : ART001, which utilizes LNP technology for targeted gene editing, is designed for the treatment of transthyretin amyloidosis (ATTR), a rare and life-threatening disease.

                            Product Name : ART001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 25, 2025

                            Lead Product(s) : ART001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            ReCode Therapeutics

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                            Peptide Therapy Summit
                            Not Confirmed

                            ReCode Therapeutics

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                            Peptide Therapy Summit
                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : RCT2100

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            ReCode’s RCT2100 Gains FDA Orphan Drug Status for Cystic Fibrosis

                            Details : RCT2100 is an inhaled mRNA-based therapy developed using our novel SORT lipid nanoparticle delivery platform. It is being developed for the treatment of cystic fibrosis.

                            Product Name : RCT2100

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 25, 2025

                            Lead Product(s) : RCT2100

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Dyne Therapeutics

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                            Peptide Therapy Summit
                            Not Confirmed

                            Dyne Therapeutics

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                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : DYNE-251

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Dyne Therapeutics Reveals Long-Term Data on DYNE-251 in Duchenne

                            Details : DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping.

                            Product Name : DYNE-251

                            Product Type : Antibody-oligonucleotide Conjugate

                            Upfront Cash : Inapplicable

                            March 16, 2025

                            Lead Product(s) : DYNE-251

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Hemab Therapeutics

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                            Peptide Therapy Summit
                            Not Confirmed

                            Hemab Therapeutics

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                            Peptide Therapy Summit
                            Not Confirmed
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                            • EDQM
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : HMB-002

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Hemab Starts Velora Pioneer Trial for Von Willebrand Disease

                            Details : HMB-002 is a monovalent human antibody, which is currently being evaluated for the treatment of von willebrand disease.

                            Product Name : HMB-002

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            February 27, 2025

                            Lead Product(s) : HMB-002

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Bionic Sight

                            U.S.A arrow
                            Peptide Therapy Summit
                            Not Confirmed

                            Bionic Sight

                            U.S.A arrow
                            Peptide Therapy Summit
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : BS01

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Bionic Sight’s BS01 Gene Therapy Receives RMAT Designation from the FDA

                            Details : FDA granted regenerative medicine advanced therapy (RMAT) designation for its investigational product BS01 for the treatment of retinitis pigmentosa patients with advanced stage vision loss.

                            Product Name : BS01

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 18, 2025

                            Lead Product(s) : BS01

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Solid Biosciences

                            U.S.A arrow
                            Peptide Therapy Summit
                            Not Confirmed

                            Solid Biosciences

                            U.S.A arrow
                            Peptide Therapy Summit
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : SGT-003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Solid Biosciences Reports Positive Data from Next-Generation Duchenne Gene Therapy

                            Details : SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding microdystrophin. It is being evaluated for duchenne muscular dystrophy.

                            Product Name : SGT-003

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 18, 2025

                            Lead Product(s) : SGT-003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Spur Therapeutics

                            United Kingdom arrow
                            Peptide Therapy Summit
                            Not Confirmed

                            Spur Therapeutics

                            United Kingdom arrow
                            Peptide Therapy Summit
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : FLT201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Spur Announces Successful End-of-Phase 2 FDA Meeting for FLT201 in Gaucher Disease

                            Details : FLT201 is an adeno-associated virus gene therapy candidate which is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

                            Product Name : FLT201

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 03, 2025

                            Lead Product(s) : FLT201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Clarametyx Biosciences

                            U.S.A arrow
                            Peptide Therapy Summit
                            Not Confirmed

                            Clarametyx Biosciences

                            U.S.A arrow
                            Peptide Therapy Summit
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : CMTX-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : KAMRA Fund

                            Deal Size : Undisclosed

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Clarametyx Biosciences Announces Investment by Kineticos AMR Accelerator Fund

                            Details : The funding aims to advance the clinical development of CMTX-101. It is being evaluated for the treatment of cystic fibrosis-related infections.

                            Product Name : CMTX-101

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            January 28, 2025

                            Lead Product(s) : CMTX-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : KAMRA Fund

                            Deal Size : Undisclosed

                            Deal Type : Funding

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