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 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 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,…..
Trend Chart On Innovative Bioindustries - July 2020, 29th FEATURE STORY GSK and CureVac announce strategic mRNA technology collaboration GSK and CureVac announced on July 20th the signing of a strategic collaboration agreement for the research, development, manufacturing and commercialisation of up to five mRNA-based vaccines and monoclonal antibodies targeting…..
Rigenerand receives regulatory approval for gene therapy production in Italy The italian biotech company Rigenerand has received received authorization from the Italian Medicine Authority (AIFA) to produce gene therapy medicinal products for clinical purposes. This approval enables Rigenerand to manufacture its own product, RR001, for the treatment of pancreatic cancer.…..
BioPharmAnalyses and OctopusyX BioConsulting are proud to announce the launch of their latest report:"AAV-based gene therapy Products: Ongoing Clinical Trials". This 205-page report provides you with a unique exhaustive overview of 116 ongoing trials for the treatment of 40 diseases (as of June 2020). More than 80 products from 49…..