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

RTTNews | 476 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 | 739 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 | 998 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 | 1004 days ago
Pound Falls As European Shares Extend Losses On Growth Worries

Pound Falls As European Shares Extend Losses On Growth Worries

The British pound weakened against other major currencies in the European session on Friday, as European shares traded lower, extending steep losses from the previous session after U.S. President Donald Trump announced significantly harsher-than-expected tariffs.
RTTNews | 4h 44min ago
DAX Down 2.2% As Stocks Extend Recent Plunge

DAX Down 2.2% As Stocks Extend Recent Plunge

After suffering its worst setback in about eight months on Thursday, the German market is deep down in negative territory again with stocks reeling under a fresh bout of selling pressure on Friday amid concerns about growth following U.S. President Donald Trump's tariff moves.
RTTNews | 4h 48min ago
Pound Falls Against Majors

Pound Falls Against Majors

The British pound weakened against other major currencies in the European session on Friday.
RTTNews | 5h 25min ago