1
Braydon Charles Guild, Michael Heartlein: mRNA for use in treatment of human genetic diseases. Shire Human Genetic Therapies, Choate Hall & Stewart, Fangli Chen, October 7, 2014: US08853377 (35 worldwide citation)

Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.


2
Braydon Charles Guild, Michael Heartlein, Frank DeRosa: mRNA for use in treatment of human genetic diseases. Shire Human Genetic Therapies, Choate Hall & Stewart, Fangli Chen, June 23, 2015: US09061021 (3 worldwide citation)

Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.


3
Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith: CFTR mRNA compositions and related methods and uses. Shire Human Genetic Therapies, Ethris, Choate Hall & Stewart, Fangli Chen, November 10, 2015: US09181321 (2 worldwide citation)

Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.


4
Frank DeRosa, Braydon Charles Guild, Michael Heartlein: Lipid-derived neutral nanoparticles. TRANSLATE BIO, Fangli Chen, Proskauer Rose, January 30, 2018: US09877919 (2 worldwide citation)

Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions. The lipids described herein are useful for example, as liposomal vehicles to facilitate the delivery of encapsulated poly ...


5
Braydon Charles Guild, Michael Heartlein, Frank DeRosa, Jerry Chi Zhang: Cleavable lipids. RaNA Therapeutics, Proskauer Rose, Fangli Chen, August 1, 2017: US09717690 (2 worldwide citation)

Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent tran ...


6
Braydon Charles Guild, Frank DeRosa, Michael Heartlein: MRNA therapy for Fabry disease. Shire Human Genetic Therapies, Choate Hall & Stewart, Fangli Chen, Meaghan E Bychowski, April 12, 2016: US09308281 (1 worldwide citation)

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.


7
Michael Heartlein, Daniel Anderson, Yizhou Dong, Frank DeRosa: Lipid formulations for delivery of messenger RNA. Shire Human Genetic Therapies, Massachusetts Institute of Technology, Proskauer Rose, April 25, 2017: US09629804 (1 worldwide citation)

The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expressio ...


8
Ferenc Boldog, Michael Heartlein: Cells for producing recombinant iduronate-2-sulfatase. Choate Hall & Stewart, October 6, 2015: US09150841 (1 worldwide citation)

The present invention provides, among other things, methods and compositions for production of recombinant I2S protein with improved potency and activity using cells co-express I2S and FGE protein. In some embodiments, cells according to the present invention are engineered to simultaneously over-ex ...


9
Frank DeRosa, Anusha Dias, Shrirang Karve, Michael Heartlein: Methods for purification of messenger RNA. Translate Bio, Fangli Chen, Proskauer Rose, December 26, 2017: US09850269

The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that ...


10
Michael Heartlein, Frank DeRosa, Anusha Dias, Shirang Karve: Methods for purification of messenger RNA. TRANSLATE BIO, Fangh Chen, Proskauer Rose, May 1, 2018: US09957499

The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized mRNA to a denaturing condition, and purifying the mRNA from the impure preparation from step (a) by tangential flow fi ...