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 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 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…..
New study expands evaluation of gene therapy for spinal muscular atrophy German researchers have just published results of a retrospective analysis of routine data collected from all patients with spinal muscular atrophy (SMA) treated with Zolgensma® (onasemnogene abeparvovec - adeno-associated viral (AAV9) vector-based gene therapy that introduces a functional copy…..
Biogen plans to build a new gene therapy manufacturing facility in Research Triangle Park Biogen, a pioneer in neurosciences, plans to build a new gene therapy manufacturing facility at its Research Triangle Park (RTP) manufacturing campuses in North Carolina to support its growing gene therapy pipeline across multiple therapeutic areas.…..
Vector-Based Gene Therapy and Gene-Editing Products Ongoing Clinical Trials Gene therapies are now a reality. They open new perspectives for the treatment and management of a wide range of rare and common diseases. We have listed ongoing clinical trials using viral vector and gene editing products to get a more…..
A new gene therapy could pave the way to the treatment of mitochondrial dysfunctions Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating dominant optic atrophy (DOA). This eye disease is characterized by degeneration of the optic nerves and primarily…..
Record financing for the regenerative medicine sector through Q3 2020 The regenerative medicine sector attracted $15.9 billion in financing through just the first three quarters of the year, shattering the previous record of $13.5 billion Global financing for the regenerative medicine and advanced therapy sector set an annual record of…..
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 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,…..
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