00:04 , Jun 25, 2019 |  BC Extra  |  Clinical News

Krystal looks to Phase III as stock soars on data for gene therapy

With its lead gene therapy showing efficacy in healing wounds in a rare skin condition, Krystal has seen its market cap multiply sixfold since its 2017 IPO as it now looks ahead to starting Phase...
00:00 , May 17, 2019 |  BC Innovations  |  Product Development

Abeona tackles gene therapy’s problems in cystic fibrosis with chimeric AAVs

As it builds up its preclinical pipeline, Abeona is switching gears to create chimeric AAV vectors that it hopes will do a better job than naturally occurring ones of dialing in the right properties for...
16:56 , Apr 19, 2019 |  BC Week In Review  |  Company News

Castle Creek gets U.S. rights to market Fibrocell's skin disease cell therapy

Castle Creek will pay $7.5 million up front for an exclusive license to commercialize Fibrocell's FCX-007 in the U.S. FCX-007 is a gene-modified autologous fibroblast that carries collagen type VII (COL7A1) to treat recessive dystrophic...
18:38 , Mar 29, 2019 |  BC Week In Review  |  Company News

ProQR spins out Wings Therapeutics

ProQR spun out its dystrophic epidermolysis bullosa business into Wings Therapeutics. Wings will continue to develop QR-313 to treat dystrophic epidermolysis bullosa patients with a mutation in exon 73. ProQR Therapeutics N.V. (NASDAQ:PRQR) will focus...
17:46 , Oct 19, 2018 |  BC Week In Review  |  Financial News

Krystal raises $60M following Phase I/II gene therapy readout

On the heels of reporting data for its topical gene therapy, Krystal Biotech Inc. (NASDAQ:KRYS) raised $60 million late Oct. 18 through the sale 3 million shares at $20 in a follow-on underwritten by Cowen,...
17:11 , Oct 19, 2018 |  BC Week In Review  |  Clinical News

Krystal's topical gene therapy meets endpoints in two patients with skin blistering disease

Krystal Biotech Inc. (NASDAQ:KRYS) reported interim data from the Phase I/II GEM trial showing that topical gene therapy KB103 met all primary efficacy and safety endpoints in two adults with recessive dystrophic epidermolysis bullosa. KB103...
20:49 , Oct 15, 2018 |  BC Extra  |  Clinical News

Krystal gains on early data for topical gene therapy in skin blistering disease

Krystal Biotech Inc. (NASDAQ:KRYS) added $4.32 (28%) to $20 on Monday when it reported interim data from the Phase I/II GEM trial showing that topical gene therapy KB103 met all primary efficacy and safety endpoints...
23:27 , Nov 30, 2017 |  BC Week In Review  |  Clinical News

Fibrocell reports Phase I/II data for FCX-007 in epidermolysis bullosa

In September, Fibrocell Science Inc. (NASDAQ:FCSC) reported interim data from a Phase I/II trial in 3 patients with recessive dystrophic epidermolysis bullosa (EB) showing that a single intradermal injection of FCX-007 led to >75% wound...
00:21 , Nov 9, 2017 |  BC Extra  |  Preclinical News

LAMB3 gene therapy successfully treats epidermolysis bullosa

In a paper published in Nature , researchers from Ruhr University Bochum and colleagues described the successful regeneration and engraftment of almost the entire epidermis of a patient with junctional epidermolysis bullosa (JEB) using laminin...
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,...