Portola Pharmaceuticals Appoints Scott Garland as President and Chief Executive Officer, Effective October 8, 2018. Portola (PTLA) appointed a new president, Mr. Scott Garland, who was Relypsa’s president and previously chief commercial officer and responsible for the integration and growth of the U.S. operations after Relypsa was acquired by Vifor Pharma Group in 2016. Prior to Relypsa, Mr. Garland was executive vice president and chief commercial officer of Exelixis, Inc., …

CRISPR Gene Editing Beyond Bringing Cures to disease-causing genes, CRISPR gene editing technology can change the way diseases are being treated and treat diseases that have yet to find a treatment. CRISPR gene editing can play a key role in solving a lot of problems that prevent breakthrough treatments and approaches from reaching their ultimate goals. A best example is chimeric antigen receptor (CAR T) Cell immunotherapy approach to …

Playing buy and sell games with the CRISPR gene editing stocks is sometimes based on scientific articles that meant to caution about possible problems or by patents that were given to some Universities and denied it to others and vice versa. Different Universities back different CRISPR gene-editing firms, which constituted a reason for the volatility of the gene-editing stocks. Finally, CRISPR Therapeutics (CRSP), Intellia Therapeutics …

The Week in Review #27 PRIORITY TOPICS: CRISPR GENE EDITING, GENETIC ENGINEERING & ANTISENSE TECHNOLOGY The choice of these topics was dictated by investors who follow the sell side preaching. These negative investors decided to cause a selloff in all kinds of therapeutics that act at the root cause of diseases, i.e., the malfunctioning genes. These therapeutics are what we call the future treatments and cures. Indeed, during …

ProhostLetter #423 TOMORROW’S THERAPEUTICS TODAY For a more or less fair evaluation of biotechnology and pharmaceutical firms, analysts currently rely for their assessment on criteria that include quarterly financial results, products’ sales revenues, and year over year growth of each and every marketed therapeutic. When it comes to clinical-stage firms, the logic implies that these criteria cannot be relied on for evaluation, as most of …

A Miracle-Like Gene-Editing That People will Sooner or Later Recognize. The Wellcome Sanger Institute’s new findings about CRISPR/Cas9 gene editing is intended to lead to further improvement in the safety of the breakthrough CRISPR gene editing procedure before it is used in clinical practice; the information is not intended to condemn it. Unfortunately, innocent investors got panicky when they heard the Wellcome Sanger findings, which …

The Week in Review #26 IN THE BIOTECH WORLD - RESEARCH - IMPROVING CRISPR GENE EDITING The problem cited with gene editing is that the editing may stay switched on after it succeeded in reaching its goals. To prevent further unwarranted mutations, scientists from the Bath and Cardiff University have invented a switch that aims at controlling protein expression in cultured cells and mouse embryos with the …

The Week in Review #25 When Stocks Plummet for No Relevant Reasons Sometimes investors need to know why some of their stocks plummet when no bad news is broadcast anywhere and when firms announce terrific news promising progressive growth. Yesterday was one of those days where reasons for stocks’ deviant, and probably unwarranted, negative performances must be identified. Let’s have a look at what happened during …

CRISPR Therapeutics, Intellia Therapeutics, and Caribou Biosciences, Inc., announced the news about the granted U.S. Patent No. 10,000,772 (“the ‘772 patent”) by the U.S. Patent and Trademark Office (USPTO). This patent covers methods of using optimized guide RNA formats (including single guide and dual guide formats) in certain environments, including eukaryotic cells (such as human, animal and plant cells). The optimized formats modify the part of …

Slashing of the CRISPR gene editing companies’ stocks, including CRISPR Therapeutics (CRSP), Editas (EDIT) and Intellia (NTLA) is caused by two studies published in Nature Medicine, which revealed that cells whose genes are edited by CRISPR/ Cas9 could develop tumors in treated patients. Nobody could blame those who sold the CRSPR gene editing stocks’ because, although many scientists believe that the cited cause can be overcome and others stated that …

CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX) announced that The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease pending the resolution of certain questions that will be provided by the FDA as part of its review of the IND. The IND was submitted to the FDA in April to support the planned initiation of a Phase …

SPARK THERAPEUTICS, SANGAMO THERAPEUTICS AND SALK INSTITUTE CURING HEMOPHILIA?   Hemophilia is an inherited bleeding disorder that causes abnormal or exaggerated bleeding and poor blood clotting. Hemophilia A and Hemophilia B are inherited in an X-linked recessive genetic pattern, so males are commonly affected while females are usually carriers of the disease. Hemophilia A is caused by clotting Factor VIII deficiency and hemophilia B (Christmas disease) is caused by Factor IX deficiency. A …

A couple of days following the news that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on Amgen’s (AMGN) product Prolia® (denosumab) for the treatment of bone loss associated with long-term systemic glucocorticoid therapy, the same Committee recommended a label variation for KYPROLIS® (carfilzomib) with an addition that includes the final overall survival (OS) data from the Phase 3 ASPIRE trial. In the ASPIRE trial, KYPROLIS …

The Week in Review #21 Part 2 THE  MARKET (2) - Our remarks in the past Week in Review included our confidence that the attempts by negative investors to drive the market crazy will not deprive the market from its innate capability to sense and react to the authentic factors it usually reacts to . . . This content is for paid subscribers. Please click here to …

The Week in Review #21 Part 1 THE MARKET - Like symphonic orchestras that bring to the listeners’ ears, minds and souls melodies composed by legendary authors, the stock market reflects what’s going on in the world, which would positively or negatively impact the lives and well being of the earth’s residents . . . This content is for paid subscribers. Please click here to subscribe or here to …

Prohost Letter #420 REAL BREAKTHROUGHS - One Has Already Been Used and the Other Has Extraordinary Promises In  the  NEWS - THE POWER OF CHECKPOINT INHIBITOR IMMUNOTHERAPY WOW It is about the checkpoint inhibitors immunotherapy, which is bringing to the world of cancer a stunning revelation of a combination treatment offering an almost cure for a lung cancer...

Alnylam (ALN) is probably experiencing a stock selloff following the announcement of successful results from Pfizer’s (PFE) drug tafamidis.    Indeed, results from Phase 3 ATT-ACT clinical trial demonstrate that tafamidis succeeded in treating Transthyretin Cardiomyopathy, a subtype of TTR amyloidosis caused by the destabilization of the protein transthyretin. Tafamidis demonstrated a statistically significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months. The drug was …

IN THE NEWS PTC THERAPEUTICS  PTC Therapeutics (PTCT) stock, which dipped yesterday, probably on news announcing a public offering of 4,000,000 shares of its common stock at a public offering price of $27.04 per share. Yesterday, in a crazy market that looked as if the bull and bear are boxers knocking each other down every 10 minutes, PTCT lost around $5. The stock seems to rebound in today’s premarket …

A False Reason for Unwarranted Selling of CRISPR/Cas9 Gene Editing Shares     CRISPRs are bacterial immune systems that contain many defense enzymes such as the Cas9 “molecular scissors,” which scientists have engineered as a powerful DNA-targeting gene-editing tool and created companies specialized in CRISPR gene editing tools. The outstanding revolutionary news, which is wrongly interpreted by Wall Street, which led to a recent selling of the shares of CRISPR gene …

Prohost Letter #418 Observing Queer Stories  PORTOLA - Portola (PTLA) has been granted approval of an important factor Xa inhibitor Bevyxxa (betrixa-­ban for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for a life-­threatening VTE . . . This content is for paid subscribers. Please click here to subscribe or here to log in.