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

RTTNews | 572 days ago
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 | 835 days ago
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 | 1094 days ago
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 | 1100 days ago
European Shares Set To Open Higher Despite Rising Trade Tensions

European Shares Set To Open Higher Despite Rising Trade Tensions

European stocks may open mostly higher on Thursday despite U.S. President Donald Trump escalating his tariff campaign, sending letters to six additional countries—Algeria, Iraq, Libya, Brunei, Moldova, and the Philippines—with fresh import duties ranging from 20-30 percent.
RTTNews | 58 minutes ago
European Economic News Preview: Germany Final Inflation Data Due

European Economic News Preview: Germany Final Inflation Data Due

Final inflation from Germany and industrial output from Italy are the top economic news due on Thursday. At 2.00 am ET, Destatis is scheduled to release Germany's final inflation data for June. Flash inflation showed that consumer price inflation eased to 2.0 percent from 2.1 percent in May.
RTTNews | 1h 19min ago
Sensex, Nifty Subdued On Trade Jitters

Sensex, Nifty Subdued On Trade Jitters

Indian shares were subdued on Thursday as U.S. President Donald Trump escalated his tariff campaign, sending letters targeting six additional countries—Algeria, Iraq, Libya, Brunei, Moldova, and the Philippines—with fresh import duties ranging from 20-30 percent.
RTTNews | 2h 11min ago
Australian Market Extends Early Gains In Mid-market

Australian Market Extends Early Gains In Mid-market

The Australian market is extending early gains in mid-market moves on Thursday, reversing the losses in the previous session, following the broadly positive cues from Wall Street overnight. The benchmark S&P/ASX 200 is moving above the 8,600 level, with gains across all sectors led by technology and mining stocks.
RTTNews | 3h 8min ago