	US 11,998,618 B2
	Frataxin expression constructs
Stephen Schauer, Rockville, MD (US); Darby Thomas, Blacksburg, VA (US); Gregory Robinson, Cambridge, MA (US); Mark Pykett, Cambridge, MA (US); Richard Thorn, Cambridge, MA (US); and Kirsten Gruis, Cambridge, MA (US)
Assigned to PRECIGEN, INC., Blacksburg, VA (US)
Filed by PRECIGEN, INC., Blacksburg, VA (US)
Filed on Dec. 17, 2020, as Appl. No. 17/125,449.
Application 17/125,449 is a division of application No. 15/807,350, filed on Nov. 8, 2017, granted, now 11,116,852.
Claims priority of provisional application 62/419,621, filed on Nov. 9, 2016.
Prior Publication US 2021/0177991 A1, Jun. 17, 2021
Int. Cl. A61K 48/00 (2006.01); A61K 38/17 (2006.01); A61P 25/00 (2006.01); C07K 14/47 (2006.01); C12N 15/86 (2006.01); C12N 15/864 (2006.01)

CPC A61K 48/0058 (2013.01) [A61K 38/1709 (2013.01); A61K 48/005 (2013.01); A61K 48/0066 (2013.01); A61K 48/0075 (2013.01); A61P 25/00 (2018.01); C07K 14/47 (2013.01); C12N 15/86 (2013.01); C12N 15/8645 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/002 (2013.01)]

21 Claims

1. A method of treating at least one symptom of Friedreich's Ataxia in a subject in need thereof, said method comprising administering to said subject a therapeutically effective amount of a composition comprising:

(a) an adeno-associated viral (AAV) vector, wherein said AAV vector comprises a nucleic acid molecule encoding human frataxin operably linked to:

a 5′UTR selected from 5U2, FTH1, GAPDH, and RPL6-5′Splice;

a 3′UTR selected from a SV40 early adenylation sequence, a SV40 late adenylation sequence, a human growth hormone polyadenylation sequence (hGHpA), and a synthetic 3′ regulatory element having a nucleic acid sequence of SEQ ID NO:7; and

a control element that directs transcription and/or translation; and

(b) a pharmaceutically acceptable excipient,

wherein the at least one symptom of Friedreich's Ataxia comprises loss of coordination in the arms and/or legs; and

wherein the administering comprises intrathecal (IT) administration, intracerebroventricular (ICV) administration, intraparenchymal administration, intracerebellar administration, or a combination thereof.