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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 Advances Inaxaplin Into Phase 2/3 Trial To Treat APOL1-Mediated Kidney Disease
Vertex Pharmaceuticals Inc. (VRTX) announced Monday that inaxaplin (VX-147) has advanced into the Phase 3 portion of the global Phase 2/3 pivotal clinical trial in APOL1-mediated kidney disease (AMKD), where a 45 mg once daily oral dose will be compared to placebo, on top of standard of care.
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374日前
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).
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745日前
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.
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1004日前
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.
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1010日前
TSX Down More Than 4% As Trade Tensions Resurface
The Canadian market is deep down in negative territory on Thursday, with investors taking some profits following the previous afternoon's sharp rally after U.S. President Donald Trump announced a 90-day pause on new "reciprocal tariffs."
Also, trade tensions between the U.S. and China weigh on sentiment.
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2時41分前
U.S. Weekly Jobless Claims Inch Up To 223,000, In Line With Estimates
A report released by the Labor Department on Thursday showed first-time claims for U.S. unemployment benefits crept slightly higher in the week ended April 5th. The Labor Department said initial jobless claims inched up to 223,000, an increase of 4,000 from the previous week's unrevised level of 219,000. The uptick by initial jobless claims came in line economist estimates.
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5時10分前
U.S. Consumer Prices Unexpectedly Edge Down 0.1% In March
The Labor Department released a report on Thursday unexpectedly showing a slight decrease by U.S. consumer prices in the month of March. The report said the consumer price index edged down by 0.1 percent in March after rising by 0.2 percent in February. Economists had expected consumer prices to inch up by 0.1 percent.
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5時13分前
Krispy Kreme Unveils Tax Break On Doughnuts For Tax Day
Doughnut maker Krispy Kreme Inc. has unveiled its Tax Day special offer on dozens of doughnuts for the third consecutive year. On the Tax Day on Tuesday, April 15, guests who purchase an Original Glazed or assorted dozen doughnuts at regular price in shop can receive a second Original Glazed dozen for only the sales tax in their state.
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6時5分前
