Vertex Receives CHMP Positive Opinion For The First CRISPR/Cas9 Gene-Edited Therapy CASGEVY

RTTNews | 398 dni temu
Vertex Receives CHMP Positive Opinion For The First CRISPR/Cas9 Gene-Edited Therapy CASGEVY

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced Friday that the European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

If approved, exa-cel would be the only genetic therapy for patients in the European Union who are 12 years of age and older with either severe SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

An approval decision by the European Commission is expected in February 2024.

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Vertex Pharma Inks Licensing Agreement To Use CRISPR Therapeutics' Gene Editing Technology

Vertex Pharma Inks Licensing Agreement To Use CRISPR Therapeutics' Gene Editing Technology

Biotechnology company Vertex Pharmaceuticals, Inc. (VRTX) and gene editing company CRISPR Therapeutics, Inc. (CRSP) announced Monday that they have entered into a new non-exclusive licensing agreement for the use of CRISPR Therapeutics' gene editing technology, known as CRISPR/Cas9, to accelerate the development of Vertex's hypoimmune cell therapies for type 1 diabetes (T1D).
RTTNews | 661 dni temu
Vertex Pharma To Acquire ViaCyte For $320 Mln In Cash

Vertex Pharma To Acquire ViaCyte For $320 Mln In Cash

Vertex Pharmaceuticals Inc. (VRTX) announced Monday that the company has entered into a definitive agreement under which Vertex will acquire privately-held biotechnology company ViaCyte for $320 million in cash.
RTTNews | 920 dni temu
Vertex Pharma Says FDA Lifts Clinical Hold On Phase 1/2 Trial Of VX-880 To Treat Type 1 Diabetes

Vertex Pharma Says FDA Lifts Clinical Hold On Phase 1/2 Trial Of VX-880 To Treat Type 1 Diabetes

Vertex Pharmaceuticals Inc. (VRTX) announced Tuesday that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia.
RTTNews | 926 dni temu
U.S. Weekly Jobless Claims Rebound To 217,000

U.S. Weekly Jobless Claims Rebound To 217,000

A report released by the Labor Department on Thursday showed first-time claims for U.S. unemployment benefits rebounded by more than expected in the week ended January 11th.
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UK Modest GDP Growth Disappoints

UK Modest GDP Growth Disappoints

The UK economy grew only marginally in November underpinned by the services sector, after two consecutive contractions, official data showed, which has strengthened the scope for more monetary policy easing from the Bank of England as inflation also softened unexpectedly at the end of 2024. Gross domestic product edged up 0.1 percent on a monthly basis in November.
RTTNews | 5g 39 minut temu
Bay Street Likely To Open Higher

Bay Street Likely To Open Higher

Canadian shares may open on a positive note on Thursday, tracking higher bullion prices and firm European stocks. The market is likely to edge higher also on hopes of some monetary easing by the Federal Reserve this year.
RTTNews | 5g 40 minut temu