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Novartis expands Zolgensma® manufacturing capacity with approval of multi-product North Carolina facility

Novartis expands Zolgensma® manufacturing capacity with approval of multi-product North Carolina facility   The FDA has granted commercial licensure approval for Novartis' Durham, N.C. site, a multi-product gene therapy manufacturing facility. This approval allows the state-of-the-art, 170,000 square-foot facility to make, test and release commercial Zolgensma®, as well as produce…..

Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology

Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology Collaboration combines Intellia’s proprietary genome editing technology platform with SparingVision’s significant ophthalmology expertise Intellia will grant SparingVision exclusive rights to its leading in vivo CRISPR/Cas9 technology for the development of ocular therapies directed to three…..

License Option Agreement with Pfizer for Voyager Therapeutic’s Next-Generation TRACERᵀᴹ AAV Capsids

License Option Agreement with Pfizer for Voyager Therapeutic's Next-Generation TRACERᵀᴹ AAV Capsids Voyager Therapeutics, a gene therapy company developing adeno-associated virus (AAV) platform technologies, announced an agreement through which Pfizer may exercise options to license novel capsids generated from Voyager’s RNA-driven TRACERTM (Tropism Redirection of AAV by Cell-type-specific Expression of…..

Positive CHMP opinion for gene therapy Libmeldy™ for the treatment of Early-Onset Metachromatic Leukodystrophy

Positive CHMP Opinion for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy® (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA,…..

G&CTI, premier think tank indépendant sur les thérapies avancées

G&CTI, premier think tank indépendant sur les thérapies avancées Dans l’environnement actuel où les thérapies géniques et cellulaires apparaissent certes comme une véritable révolution médicale, mais où de nombreux défis éthiques, sociétaux, politiques, économiques se posent, la quête de réponses opérationnelles s’impose ! L’ensemble des acteurs (sociétés savantes, patients, industriels,…..