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
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 | 52 minutes ago
Asian Markets Track Wall Street Higher

Asian Markets Track Wall Street Higher

Asian stock markets are trading mostly higher on Thursday, following the broadly positive cues from Wall Street overnight, as traders are optimistic about an end to the tariff uncertainty after US President Donald Trump made it clear that he will not extend the August 1 deadline for trade deals, though he gets aggressive with tariff imposition.
RTTNews | 57 minutes ago
Sensex, Nifty Set For Muted Open

Sensex, Nifty Set For Muted Open

Indian shares look set to open on a flat note Thursday as investors watch the latest developments on the trade front, react to the latest FOMC meeting minutes and look for direction from Q1 business updates from prominent companies.
RTTNews | 1h 43min ago
Indonesia Stock Market May Extend Winning Streak

Indonesia Stock Market May Extend Winning Streak

The Indonesia stock market has moved higher in three straight sessions, gathering almost 80 points or 1.2 percent along the way. The Jakarta Composite Index now sits just above the 6,940-point plateau and it may add to its winnings again on Thursday.
RTTNews | 2h 44min ago