Tag: Spark Therapeutics (ONCE)

The Value of the Biotech Companies Continued

The Value of the Biotech Companies Continued

Prohost Letter #435 The Value of the Biotech Companies A Continuation of Prohost Letter #434 A Recap The biotech near and long-term values, we insist, must be based on the firms’ scientific capabilities, products’ potentials and financial results. In the past months the stocks evaluations by bloggers, and some assessors, have been tailored to mislead investors into serving the purposes of the manipulators’ weird agendas. …
Gene Therapy and Other Treatments That Work at the Genetic Level

Gene Therapy and Other Treatments That Work at the Genetic Level

Gene Therapy Stocks Experiencing Selloffs  Every investor in the biotech sector must have experienced selloffs of the stocks from companies specialized in gene therapy, gene editing, antisense, RNAi and mRNA therapies. Recently, bluebird bio’s (BLUE) stock was devastated. The selloffs occurred for many reasons, most of . . . This content is for paid subscribers. Please click here to subscribe or here to log in.
bluebird bio Announces a European Conditional Approval of Its Gene Therapy ZYNTEGLO™ for Transfusion-Dependent β-Thalassemia  

bluebird bio Announces a European Conditional Approval of Its Gene Therapy ZYNTEGLO™ for Transfusion-Dependent β-Thalassemia  

Zynteglo™ from bluebird bio Gets European Conditional Approval European marketing authorization for Zynteglo follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program. Zynteglo is bluebird bio’s first gene therapy to gain regulatory approval. Gene therapy is, indeed, moving towards the clinic to provide cures; to save people from an early death from …
Gene therapy: Speeding down the road towards offering cures. A small firm might have some good news

Gene therapy: Speeding down the road towards offering cures. A small firm might have some good news

We love to read news about gene therapy as it is the only possibility for curing diseases that no known treatment can halt or delay their progression. The attempts to use gene therapy failed ambitious researchers for several decades before the revolution that led to understanding the genome and even decades  more after the publishing of the genomic map that actually started the real revolution. …
Good news and setbacks during the departed week

Good news and setbacks during the departed week

The Week in Review #38 SYNOPSIS Good News and Setbacks During the Departed Week from the Prohost Portfolio FIRMS WITH GOOD NEWS SPARK THERAPEUTICS The most important happening, with the most exciting positive impact, has come this time from our picked gene therapy company, Spark Therapeutics (ONCE). It was electrifying news, which announced acquisition by Roche (RHHBY) of Spark Therapeutics; at a price of $114.50 …
Spark Therapeutics to be acquired by Roche at $113.47 UP $61.91 

Spark Therapeutics to be acquired by Roche at $113.47 UP $61.91 

We congratulate our subscribers who kept Spark Therapeutics (ONCE) in spite of the stock’s kind of paralysis for no serious reasons. Spark, a fully integrated, commercial gene therapy company, announced today that it has entered into a definitive merger agreement with Roche (RHHBY). The firm is to fully acquire Spark Therapeutics at a price of $114.50 per share, in an all-cash transaction. This move represents  a total equity value …
Successful results were announced by uniQure for its gene therapy, AMT-061, on hemophilia B patients

Successful results were announced by uniQure for its gene therapy, AMT-061, on hemophilia B patients

The United States FDA granted Breakthrough Therapy Designation to AMT-061. The European Medicines Agency gave it access to the Priority Medicine (PRIME) regulatory initiative. Gene therapy firm, uniQure (QURE), announced updated clinical data in patients treated in the ongoing Phase 2b study of AMT-061; an investigational AAV5-based gene therapy containing a patent-protected  FIX9-Padua (Factor 9) for the treatment of patients with severe and moderately severe hemophilia B. The announced data, which …
When common sense fails to explain market performance

When common sense fails to explain market performance

Prohost Letter #425 When Common Sense Fails to Explain the Market Performance It is not unusual for the stock market to be in a selloff mood for a few days. However, in the last couple of months, the market unexpectedly plunged deep into the ocean of the unknown. The exaggerated volatility was nerve-wracking for professional analysts. This is especially true for candid analysts who base …
The molecule that enhanced the T Cells cancer treatment efficacy while preventing autoimmune diseases

The molecule that enhanced the T Cells cancer treatment efficacy while preventing autoimmune diseases

Yesterday, the Stock Market skyrocketed and with it a significant number of Prohost Picks that we keep reminding ourselves are undervalued. The outperformance unveiled the interred strength of firms such as: Illumina (ILMN), Amgen (AMGN) and Vertex (VRTX), the gene therapy firms: RegenxBio (RGNX) and Spark (ONCE), and the gene editing firms: Crispr (CRSP), Intellia (NTLA) and Editas (EDIT), as well as others in the Prohost Portfolio. The rebounding in the …
We call it the bad market

We call it the bad market

The Week in Review #33 WE CALL IT THE BAD MARKET The Federal Reserve’s raising of the interest rate does not and should not mean a demise of the companies trading on the NASDAQ and other stock exchanges. The biotech group of firms with strong scientific fundamentals will not stop innovating and bringing breakthroughs to millions of people who are desperate for them if an …
Following the Stars. Less Science and More Logic

Following the Stars. Less Science and More Logic

Prohost Letter #424 Following the Stars Less Science and More Logic We reiterate that while a human star is born every once in a while on Earth, several stars born daily in research labs may be designated every other day by the FDA and other health regulatory agencies around the world. These agencies call these potential stars’ breakthrough drugs, but not all the molecules designated …
Spark Therapeutics: An encouraging attempt to dethrone enzyme replacement as the standard of care treatment for Pompe Disease.

Spark Therapeutics: An encouraging attempt to dethrone enzyme replacement as the standard of care treatment for Pompe Disease.

On Saturday, October 6, 2018, Spark Therapeutics (ONCE) presented positive data from a preclinical trial with SPK-3006 for the treatment of Pompe disease in an oral presentation at the 23rd International Congress of the World Muscle Society in Argentina. SPK-3006 is an investigational liver-directed adeno-associated viral (AAV) gene therapy being developed for Pompe disease. Pompe disease is caused by the buildup of a complex sugar, glycogen, in the body’s cells. It is …
Starting the gene therapy chapter in the book of the biotech revolution

Starting the gene therapy chapter in the book of the biotech revolution

WE ARE LIVING AN HISTORIC BIOLOGICAL REVOLUTION. Unfortunately, the undeniably outstanding news has not yet penetrated the ears of many investors. They are still falling victim to the stock market traders’ games and to critics’ and bloggers’ magnifications or fabrications of negative news. Many investors end up selling their thoughtfully chosen highly scientific biotech firms to regret later on what they have done when their …
Bombarding future treatment approaches

Bombarding future treatment approaches

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 …
Spark Therapeutics: A usual reaction to SPK-8011 results should have been a celebration

Spark Therapeutics: A usual reaction to SPK-8011 results should have been a celebration

When an investigational gene therapy gets excellent results, the usual reaction towards the developing firm is a celebration. This is especially true when the developing firm is Spark Therapeutics (ONCE), which has alreadyvalidated its gene therapy approach through the approval of its product LuxturnaTM (voretigene neparvovec-rzyl).         In its quarterly financial report, Spark said that 12 participants in Phase I/II trial investigating SPK-8011 for hemophilia A
Victims of fear and misunderstanding

Victims of fear and misunderstanding

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 …
Revisiting Biotech Companies We Highlighted

Revisiting Biotech Companies We Highlighted

Prohost Letter #421 PRIVATE  LESSONS - Stock prices of publicly-­traded biotech companies oscillate for the same reasons other industries’ stock prices swing all day long. The fluctuations are the outcome of investors’ and daily traders’ unrelenting buying and selling . . . This content is for paid subscribers. Please click here to subscribe or here to log in.
Cures for Hemophilia A and B? See Also: Why EXEL Was a Loser Yesterday

Cures for Hemophilia A and B? See Also: Why EXEL Was a Loser Yesterday

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 …
AveXis Drug’s Data for Spinal Muscular Atrophy Highlights Gene Therapy

AveXis Drug’s Data for Spinal Muscular Atrophy Highlights Gene Therapy

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 Year in Review Part 2

The Year in Review Part 2

Prohost Letter #416 Part 2 2017 - A YEAR OF BREAKTHROUGH TECHNOLOGIES, BREAKTHROUGH PRODUCTS, IMPROVEMENTS ON PROMISING BREAKTHROUGH TREATMENTS, DECIPHERING IMPORTANT LIFE SCIENCE MYSTERIES, PINPOINTING BIOLOGICAL PATHWAYS FOR TREATMENT RESISTANCE, MIRACLE CURES, AND… A LOT OF DISTORTED NEGATIVE INTERPRETATIONS - Prioritizing our choices of breakthroughs has been a dilemma . . . This content is for paid subscribers. Please click here to subscribe or here to log …