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Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amplo Biotechnology \/ Andelyn Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Amplo Biotechnology \/ Andelyn Biosciences"}]

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                            Lead Product(s) : AMP-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Andelyn Biosciences

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                            Andelyn and Amplo Partner to Manufacture Novel Gene Therapies

                            Details : The partnership centers on manufacturing clinical-grade AAV vectors using Andelyn's suspension AAV Curator® platform to support Amplo’s NMJ-focused gene therapy candidates, including AMP-101.

                            Product Name : AMP-101

                            Product Type : Protein

                            Upfront Cash : Undisclosed

                            August 21, 2025

                            Lead Product(s) : AMP-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Andelyn Biosciences

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : National Cancer Institute

                            Deal Size : Undisclosed

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Endure Receives SBIR Phase I Grant to Advance Engineered Native Bacteria Therapy

                            Details : The funding will support the development of a novel Engineered Native Bacteria (ENB) candidate for the treatment of Familial Adenomatous Polyposis (FAP).

                            Product Name : Undisclosed

                            Product Type : Microorganism

                            Upfront Cash : Undisclosed

                            August 12, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : National Cancer Institute

                            Deal Size : Undisclosed

                            Deal Type : Funding

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                            03

                            BioMarin Pharmaceutical

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            BioMarin Pharmaceutical

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                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : BMN 390

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            BioMarin Axes Pre-Clinical PKU Drug, Citing Underwhelming Immunogenicity

                            Details : BMN 390 was a preclinical enzyme therapy for phenylketonuria (PKU), designed to break down phenylalanine with reduced immunogenicity by using a novel non-PEG polymer for fewer reactions.

                            Product Name : BMN 390

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            August 05, 2025

                            Lead Product(s) : BMN 390

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Rallybio

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Rallybio

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                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : REV102

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Recursion Pharma

                            Deal Size : $25.0 million

                            Deal Type : Agreement

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                            Rallybio Sells Interest in REV102 Program to Recursion Pharmaceuticals

                            Details : Rallybio sell its interest in REV102, an ENPP1 inhibitor in preclinical development for the treatment of patients with hypophosphatasia (HPP) to Recursion.

                            Product Name : REV102

                            Product Type : Other Small Molecule

                            Upfront Cash : $7.5 million

                            July 08, 2025

                            Lead Product(s) : REV102

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Recursion Pharma

                            Deal Size : $25.0 million

                            Deal Type : Agreement

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                            05

                            Cure Rare

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Cure Rare

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                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : CRD-003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Cure Rare Disease Gets FDA Feedback on Limb-Girdle 2i/R9 Gene Therapy Program

                            Details : The LGMDR9 program, known as CRD-003, utilizes a novel, liver de-targeting, muscle tropic capsid. It is being evaluated for the treatment of Limb-Girdle Muscular Dystrophy type 2I/R9.

                            Product Name : CRD-003

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            June 23, 2025

                            Lead Product(s) : CRD-003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Repair Biotechnologies

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Repair Biotechnologies

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                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : REP-0003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Repair Biotech Wins FDA Orphan Drug Tag for Homozygous Familial Hypercholesterolemia

                            Details : REP-0003 is a cutting-edge messenger RNA therapy that clears harmful excess free cholesterol inside cells while preserving essential cholesterol, for homozygous familial hypercholesterolemia.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            May 19, 2025

                            Lead Product(s) : REP-0003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            IPS Heart

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed

                            IPS Heart

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

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            IPS HEART Granted Orphan Drug Designation in the EU for Becker Muscular Dystrophy

                            Details : GIVI-MPCs derived from pluripotent stem cells form new skeletal muscle with 100% full-length human dystrophin for DMD and BMD.

                            Product Name : GIVI-MPC

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 14, 2025

                            Lead Product(s) : GIVI-MPC

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Airna

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Airna

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                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : AIR-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Venrock Healthcare Capital

                            Deal Size : $155.0 million

                            Deal Type : Series B Financing

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                            Airna Raises $155 Million Series B Financing for Alpha-1 Antitrypsin Deficiency

                            Details : The financing aims to advance company's RNA editing product candidate AIR-001, which is being evaluated for the treatment of Alpha-1 antitrypsin deficiency.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            April 01, 2025

                            Lead Product(s) : AIR-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Venrock Healthcare Capital

                            Deal Size : $155.0 million

                            Deal Type : Series B Financing

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                            09

                            Prime Medicine

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                            SCI Medicinal Chemistry
                            Not Confirmed

                            Prime Medicine

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

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Prime Medicine Unveils Program for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

                            Details : This program leverages the Company’s universal liver lipid nanoparticle (LNP) to edit the E342K (Pi*Z) mutation in the SERPINA1 gene, the prevalent disease-causing mutation in AATD.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            March 18, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Porosome Therapeutics

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed

                            Porosome Therapeutics

                            U.S.A arrow
                            SCI Medicinal Chemistry
                            Not Confirmed
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                            Lead Product(s) : CF PT-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Orphan Drug Designation to Revolutionary Cystic Fibrosis Therapy

                            Details : CF PT-101 is a porosome reconstitution therapy, which is currently being evaluated for the treatment of cystic fibrosis.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 25, 2025

                            Lead Product(s) : CF PT-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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