It has been demonstrated that proliferation and expansion of T lymphocytes in ADA-SCID
is prevented via A2a receptor activation.
The clinical data we've seen to date with Orchard's therapy for ADA-SCID
is exceptional, and the company has assembled and impressive R&D pipeline to apply the approach more widely.
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) today announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID
(Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency).
Therapeutic areas included ZFN-based approaches to infectious diseases such as HIV/AIDS and monogenic diseases such as hemophilia, ADA-SCID
, retinal neurodegeneration and epidermolysis bullosa.
It was reported on Friday that the grant will fund a new clinical trial for Severe Combined Immunodeficiency caused by adenosine deaminase deficiency, commonly known as ADA-SCID
or "bubble baby" disease.
GlaxoSmithKline today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), in conjunction with the Committee for Advanced Therapies (CAT), has issued a positive opinion recommending marketing authorisation for Strimvelis to treat patients with a very rare disease called ADA-SCID
(severe combined immunodeficiency due to adenosine deaminase deficiency).
The European Commission has approved Strimvelis, an ex-vivo stem cell gene therapy intended for the treatment of a very rare disorder called ADA-SCID
, or severe combined immunodeficiency due to adenosine deaminase deficiency, it was reported yesterday.
5 August 2011 - Italy-based biotechnology company Molecular Medicine SpA (MolMed) (BIT:MLM) announced today that it has entered into an agreement with UK-based healthcare group GlaxoSmithKline (LON: GSK), under which MolMed will develop a production process for an investigational gene therapy for ADA-SCID
(Adenosine Deaminase Deficiency - Severe Combined Immune Deficiency).