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Vertex has acquired a positive positioning for KAFTRIO ®

Vertex Pharmaceuticals (VRTX:NASDAQ) is an American biopharmaceutical company headquartered in Boston, Massachusetts. It was one of the first biotechnology companies to use an explicit strategy of rational drug design without chemistry. The company has three research facilities, in Sandiego, California, and in Milton Park and Oxfordshire, England. Vertex was founded in 1989 by Joshua Boger and Kevin J. Kinsella to «transform the way we treat serious diseases. Until 2004, the company focused primarily on viral infections, inflammatory, autoimmune diseases and cancer. In 2009, the company had about 1,800 employees. In 2019, there were about 2,500 employees. Since the end of 2011, Vertex has been ranked among the top 15 best performing companies in the Standard & Poor’s 500 . Vertex stock has increased 250% over the same period. In January 2014, Vertex completed its move from Cambridge, Massachusetts to Boston, Massachusetts, and settled into a new $800 million complex. This is located on the waterfront in South Boston. In June 2019, Vertex Pharmaceuticals (VRTX:NASDAQ) announced that it will acquire Exonics Therapeutics for $1 billion and partner with CRISPR Therapeutics to advance its development of treatments for Duchenne disease, a so-called muscular dystrophy, and myotonic dystrophy type 1.

Vertex Pharmaceuticals Incorporated(Nasdaq: VRTX) announced that the European Medicines Agency (CHMP) (Committee for Medicinal Products for Human Use ) has adopted a positive opinion on the approval of KAFTRIO ® , for the treatment of cystic fibrosis in children aged 2 to 5 years. The first of these medicines, KALYDECO ® (ivacaftor), was approved in 2012. A new drug, KAFTRIO ®, is currently approved for children aged 1 month and older.

Vertex researchers have also won a breakthrough prize for life science, among others. Chief Scientific Officer and Executive Vice President of Global Research David Altshuler, M.D., Ph.D. said «The research teams led by Paul, Fred and Sabine have discovered the first and only drug that addresses the underlying cause of cystic fibrosis. This remarkable effort required the discovery of three new mechanisms of action, leading to the first disease-modifying therapies that work by restoring the function of a misfolded protein.» «This award recognizes both the dedication and creativity of thousands of researchers, as well as the development, access to patients, and tireless staff who have worked to bring medicines to people with CF around the world.»

The company also recently reported financial results for the second quarter of 2023

 The second quarter of 2023 marked another period of significant progress for the business. Cystic fibrosis drugs are reaching more and more patients around the world than ever before. Vertex Pharmaceuticals (VRTX:NASDAQ) is also moving other programs with clinical trials into the final phase, making rapid progress in the development of additional drugs.

Tržby společnosti Vertex Pharmaceuticals (VRTX:NASDAQ) v porovnání se stejným čtvrtletím předchozího roku vzrostly o 14% na 2,49 miliardy USD. Společnost také zvýšila náklady na výzkum a vývoj z 877 milionu USD na 1,2 miliardy USD. Čistý zisk se meziročně zvýšil o 13% na především díky silnému růstu výnosů.

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CABALETTA BIO, INC. has developed a revolutionary drug

Cabaletta Bio (NASDAQ:CABA) was founded in 2017.  The company is headquartered in Philadelphia, PA, USA. The company is focused on developing T-cell therapeutics for the treatment of autoimmune diseases. The company collaborates with a number of professional institutions such as the University of Pennsylvania, Children’s Hospital of Philadelphia, and biotechnology company IASO Bio.

Cabaletta Bio announced via press release that it has developed a groundbreaking drug, CABA-201, for the treatment of autoimmunity through fully human chemical active receptors. The company has also extended an agreement with WuXi Advanced Thzerapes to manufacture this new drug, CABA-201. This extension of the agreement facilitates the preparation of CABA-201 for commercial use and enables the treatment of patients in several planned clinical trials. This extension of the CABA-201 collaboration builds on an existing manufacturing agreement to supply MuSK-CAART (a drug for an autoimmune disease affecting neuromuscular junctions) for clinical trials.

President of Cabaletta Bio, Gwendolyn Binder said «For the past two years we have had a successful collaboration with WuXi ATU (a biopharmaceutical company that is involved in molecular research, development, manufacturing and biological research, gene therapy testing of medical products) to produce novel cell therapies in a GMP compliant manner. «Based on this collaboration, we have decided to expand our partnership to include WuXi ATU as the manufacturer of our CABA-201 (fully human, chemical, antigen receptor) clinical programs.» These facts have attracted the attention of multinational investment corporations and private investors to buy shares of Cabaletta Bio (NASDAQ:CABA) in particular, for which they expect strong growth in the share price, for which an average target price of $24.33 per share has been set for the short- to medium-term investment horizon.