LEXINGTON, Mass. and AMSTERDAM – uniQure NV (NASDAQ: QURE), a gene therapy division, has reached an agreement with the US FDA on one of the best results for the experimental gene therapy AMT-130 for the treatment of Huntington’s disease. It is a milestone on the road to a new treatment option for patients with this genetic neurodegenerative disease.


Based on the Vereinbarung, data from the phase I/II study can be used in the comparison of an external control group as a primary basis for the Einreichung and Zulassungsantrags (BLA) for AMT-130. It may be that the Notnauwkeit sisterätzlicher Vorstudien eliminates. The FDA encourages this because the Unified Huntington’s Disease Rating Scale (cUHDRS) as an intermediate clinical endpointer could cause a reduction of neurofilament light chain proteins (NfL) in beverage (CSF) as a disruptor to the therapeutic benefits.


In May 2024, the FDA will grant Regenerative Medicine Advanced Therapy (RMAT) status for AMT-130 and there may be a chance that this treatment will undergo an unfunded medical investigation into Huntington’s Disease treatment. Zwischenergebnisse vom July 2024 deuteten darauf hi, the AMT-130 das Fortschreiten der Krankheit was based on cUHDRS-verlangsamen and the NfL-Spiegel im Liquor reduzieren könnte.


It is one of the most versatile, dose-escalating Phase I/II studies that have used a Sicherheit, Verträglichkeit and exploratory Wirksamkeit of AMT-130. The US trial has completed 26 patients with Huntington’s disease manifestation, while the European Phase Ib/II trial has completed 13 patients. A sister with a short summary would not be able to use the therapy in combination with immunosuppression.


The Huntington’s disease problem has affected about 70,000 people in the US and Europe, who no longer have any therapy for the treatment of the Australian language or for the development of the Fortschreiten-Verfügbar sind. UniQure factory, in the first half of the 2025s, continues with the FDA to investigate, a statistical analysis plan and technical research for the BLA studies into the future.


This expansion takes place at a press conference of uniQure Inc. and reflects the fortress of the Unternehmens at the Entwicklung of AMT-130 as potential management for the Huntington’s disease more broadly.


In others, uniQure NV has conducted a Phase I/II Study for its experimental Treatment AMT-162 with Treatment of ALS, performed by SOD1-Mutatie. The FDA has achieved Orphan Drug designation and Fast-Track designation is available. Darüber has treated the FDA Gene Therapy experimenter AMT-191 with Fabry-Krankheit orphan drug designation.


Zusätzlich hat das Unternehmen bedeutende Entwicklungen in seinem clinical Portfolio verzeichnet. UniQure’s AMT-130 gene therapy for Huntington’s disease is a sweetly encouraging dating.


Raymond James has received the Beobachtung van een UnieQure assessment and received an “Outperform” rating. This is the robust clinical portfolio of the university and the financial state pasture reformed until the end of 2027. HC Wainwright and Stifel have fallen from their Kaufempfehlung for a unique challenge.


Abschließend hat uniQure eine bedeutende decisive Umstrukturierung duchgeführt, einschließlich een 65-prozentigen Reduzierung seiner Belegschaft und des Verkaufs seiner Productionsanlage in Lexington, Massachusetts. Genezen has developed a number of commercial gene therapy businesses in Lexington and says these are global lung cancer opportunities for gene therapies. It is a fact that the young people started in the Unternehmensabläufen.


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