19:35 , Oct 19, 2018 |  BC Week In Review  |  Company News

Sarepta gets gene therapy candidate, Lysogene extends runway

Lysogene S.A. (Euronext:LYS) granted Sarepta Therapeutics Inc. (NASDAQ:SRPT) exclusive, ex-European rights to LYS-SAF302, an adeno-associated virus (AAV)-mediated gene therapy. Sarepta will pay $15 million up front and make a $2.5 million equity investment in Lysogene,...
15:32 , May 25, 2018 |  BC Week In Review  |  Clinical News

Abeona reports updated data for MPS IIIA gene therapy

Abeona Therapeutics Inc. (NASDAQ:ABEO) said ABO-102 led to significant dose- and time-dependent reductions in CSF and urine heparan sulfate levels through 18 months in a Phase I/II trial to treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo...
23:26 , May 18, 2018 |  BC Extra  |  Clinical News

Abeona reports updated data for MPS IIIA gene therapy

Abeona Therapeutics Inc. (NASDAQ:ABEO) said ABO-102 led to significant dose- and time-dependent reductions in CSF and urine heparan sulfate levels through 18 months in a Phase I/II trial to treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo...
21:53 , Dec 1, 2017 |  BC Week In Review  |  Clinical News

Abeona reports top-line Phase I/II data for ABO-102 in mucopolysaccharidosis IIIA

In October, Abeona Therapeutics Inc. (NASDAQ:ABEO) reported top-line data from the first cohort of 3 patients with mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome) in a Phase I/II trial showing that IV ABO-102 (AAV-SGSH)...
00:34 , Oct 20, 2017 |  BC Week In Review  |  Financial News

Abeona raises $92M in follow-on

Gene therapy play Abeona Therapeutics Inc. (NASDAQ:ABEO) raised $92 million through the sale of 5.8 million shares at $16 in a follow-on underwritten by Jefferies, RBC Capital Markets, Cantor Fitzgerald, FBR Capital Markets, H.C. Wainwright,...
21:42 , Oct 17, 2017 |  BC Extra  |  Financial News

Abeona raises $80M in follow-on

Gene therapy play Abeona Therapeutics Inc. (NASDAQ:ABEO) raised $80 million through the sale of 5 million shares at $16 in a follow-on underwritten by Jefferies, RBC Capital Markets, Cantor Fitzgerald, FBR Capital Markets, H.C. Wainwright,...
19:22 , Jul 28, 2017 |  BioCentury  |  Regulation

Ganging up in rare disease

A joint proposal from EMA and FDA that encourages companies to join multidrug platform trials for pediatric Gaucher’s disease came too late to serve its original purpose. But the proposal could provide a template for...
19:45 , Apr 21, 2017 |  BioCentury  |  Emerging Company Profile

Orchard’s pickings

Orchard Therapeutics Ltd. ’s autologous stem cell therapies could improve upon more advanced cell and gene therapies that also were designed to produce durable responses by restoring production of missing enzymes in rare genetic diseases....
11:38 , Dec 1, 2016 |  BC Week In Review  |  Clinical News

ABO-102: Interim Ph I/II data

Interim data from 3 patients with MPS IIIA in an open-label, U.S. Phase I/II trial showed that a single IV injection of 5x10 12 vg/kg ABO-102 was well tolerated with no treatment-related serious adverse events...
19:57 , Nov 4, 2016 |  BC Week In Review  |  Clinical News

SHP610: Development discontinued

Shire said it will discontinue development of SHP610 after it missed the primary endpoint in an open-label, international Phase IIb trial. The company said SHP610 failed to slow cognitive decline in pediatric patients. Shire plc...