Modernatx, Inc.

Fundacion Para La Investigacion Medica Aplicada

Phoenix Tissue Repair, Inc.

Lin Tung Guey

Kerry Benenato

Stephen G Hoge

Vladimir Presnyak

Ellalahewage Sathyajith Kumarasinghe

Iain McFadyen

Paolo Martini

Staci Sabnis

Xuling Zhu

Jingsong Cao

Pedro Berraondo Lopez

Igor Quinones-Garcia

Antonio Fontanellas Roma

Bing He

Sujit Basu

Lei Jiang

Matias Antonio Avila Zaragoza

Kris Lowe

Vinh Nguyen

Amey Bandekar

Iain Mcfadyen

The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).

Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria

The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 ('CTLN2'). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.

Polynucleotides encoding Citrin for the treatment of Citrullinemia type 2

The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the &#x3b1;-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

Polynucleotides encoding &#x3b1;-galactosidase A for the treatment of Fabry disease

Polynucleotides encoding citrin for the treatment of Citrullinemia type 2

The present disclosure relates to compositions comprising collagen 7 and methods of using the same to treat DEB.

Collagen 7 compositions and methods of using the same

Polynucleotides encoding alpha-galactosidase A for the treatment of Fabry disease

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Lexington, MA, United States of America

Lin Tung Guey