BioCentury | Apr 20, 2020
Finance

French VC Sofinnova expanding start-up ecosystem in Italy with new local fund’s first investments

With the first three investments from a new fund launched in partnership with Italian charity Fondazione Telethon to invest in therapies for rare genetic diseases, Sofinnova Partners is aiming to build and expand the country’s...
BioCentury | Dec 6, 2019
Targets & Mechanisms

A view of immune reprogramming from ASH

...which can be disrupted by blocking iron trafficking ( No. 3059 ). Targets ADA2 - Adenosine deaminase 2 (ADA2)...
BioCentury | Sep 4, 2019
Company News

Novartis’ early collaboration with NICE key to rapid response on Luxturna

In one of NICE’s fastest draft recommendations for a new therapy on the NHS, the HTA found gene therapy Luxturna from Novartis to be cost-effective to treat a rare genetic retinal disorder. Novartis’ offer of...
BioCentury | Jun 17, 2019
Company News

Trial data suggest 80% of Zynteglo patients could meet milestones in outcomes-based pricing model

The most recent long-term data for bluebird bio's Zynteglo β thalassemia gene therapy show that about 80% of patients could hit payment milestones based on details the biotech disclosed about its outcomes-based pricing model on...
BioCentury | Dec 9, 2017
Preclinical News

A faster method for harvesting HSCs for transplantation

Researchers report a new two-drug regimen could speed up harvesting of hematopoietic stem cells for transplantation and yield a population of cells that engraft more efficiently than those produced by standard regimens. In a paper...
BioCentury | Oct 27, 2017
Company News

NICE recommends GSK's Strimvelis for ADA-SCID

The U.K.'s NICE issued draft guidance recommending gene therapy Strimvelis (GSK2696273) from GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) for whom no suitable HLA-matched related stem cell...
BioCentury | Oct 22, 2017
Company News

NICE recommends Strimvelis for ADA-SCID

The U.K.'s NICE issued draft guidance recommending gene therapy Strimvelis (GSK2696273) from GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) for whom no suitable HLA-matched related stem cell...
BioCentury | Jul 17, 2017
Company News

Priority Review for Spark's ophthalmic gene therapy

Spark Therapeutics Inc. (NASDAQ:ONCE) said FDA accepted and granted Priority Review to a BLA for gene therapy Luxturna voretigene neparvovec (AAV2-hRPE65v2) to treat vision loss due to confirmed biallelic retinal pigment epithelium-specific protein 65kDa (RPE65)-mediated...
BioCentury | Apr 21, 2017
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....
BioCentury | Dec 31, 2016
Finance

Delivering takeouts

BioCentury’s 25th annual Buyside View finds biotech investors focusing on mid-cap names with late-stage or marketed products that could become M&A targets. “The main theme playing at the moment is investors like us are looking...
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