Tag: Spark Therapeutics (ONCE)

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 …
The Year in Review Part 1

The Year in Review Part 1

Prohost Letter #416 Part 1 The Year In Review - 2017 Unheard of Programs - AN AWESOME YEAR for the biotech sector regarding its accomplishments. Many of the accomplished programs were unheard of regarding the degree of their positive impact on medical practice. Unfortunately, many of these accomplishments, which included the making of new discoveries, the creation of novel advanced technologies and the design of …
Juno and Spark: On the Road Towards Ambitious Goals

Juno and Spark: On the Road Towards Ambitious Goals

Juno Therapeutics (JUNO) is one of the casualties that occurred during the 59th American Society of Hematology (ASH) Annual Meeting. The stock experienced undeserved selloff except for the fact that it had a deserved rally that we believe will remain as long as the firm is spending effort towards improving its cancer treatment. This cancer treatment is the  living genetically engineered chimeric antigen receptor (CAR) T cell expected …
Last Week’s Promising News

Last Week’s Promising News

The Week in Review #13 From the Prohost Portfolio - 1. Betting on the technology - HALOZYME - As expected, the pulse of fat agreements with HALO is accelerating . . . This content is for paid subscribers. Please click here to subscribe or here to log in.
The Week in Review #11

The Week in Review #11

The Week in Review #11 A New Era - Faster than the speed of light, the management of diseases is shifting from fast improvement to a total gigantic unprecedented change. Some of the conquering approaches towards providing long-­lasting cures look as if the outcome of magic rather than mere science . . . This content is for paid subscribers. Please click here to subscribe or here to log …
The FDA New Guidelines to Speed the Approval of Gene Therapy. See Also: The Gene Editing Effect

The FDA New Guidelines to Speed the Approval of Gene Therapy. See Also: The Gene Editing Effect

Important news has come from the FDA, which issued new guidelines to speed the introduction of treatments with human cells and tissues, including gene therapy. Cracking down on rogue clinics offering dangerous or unproven versions of gene therapy or stem cell therapy is also included in the report. Scott Gottlieb, the commissioner of the FDA stated that regenerative medicine and other important gene therapy treatments …
Spark Therapeutics: Making History

Spark Therapeutics: Making History

Yes, indeed, the FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease LUXTURNA has the potential to be the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the United State First adeno-associated viral (AAV . . . This content is for paid subscribers. Please click here to subscribe …
Ardelyx’s Exciting News. Spark Therapeutics: Facing the Moment of Thrilling Truth

Ardelyx’s Exciting News. Spark Therapeutics: Facing the Moment of Thrilling Truth

Good news emanating from late phase trials by development-stage firms is always refreshing for Prohost, especially when the news brings hope for the suffering patients whose diseases are not adequately responding to existing treatments. Today GOOD news is coming from late Phase trials announcing that a product called Tenapanor has met all its Aprimary and secondary endpoints for irritable colon syndrome with constipation (IBS-C), which …
Prohost Letter #411

Prohost Letter #411

Prohost Letter #411 SOME STOCK RALLYING NEWS - Under normal conditions, some good news should rally the stocks of the firms that the news belong to them. During the past six months, Amgen (AMGN) announced a series of good news that seem to have fallen on deaf ears. Yesterday Amgen announced some positive results from three approved and one experimental products – all the news …