Tag: Spark Therapeutics (ONCE)

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 …
Prohost Letter #410

Prohost Letter #410

Prohost Letter #410 JUNE PDUFA DAYS This month, the FDA will decide on the approvals of drugs belonging to companies that Prohost has picked for investment. Usually, granting approvals rallies the stocks of the drugs’ developing firms. Sending Complete Response Letters for demands that might delay approvals would cut a substantial percentage of the stocks’ values, while rejections would cause the stocks to falter. Let’s …
Editas, Adverum and Spark: Novel Gene Editing and Viral Vectors Make Gene Therapy Achievable

Editas, Adverum and Spark: Novel Gene Editing and Viral Vectors Make Gene Therapy Achievable

Gene Editing EDITAS MEDICINE CRISPR gene editing firm Editas Medicine (EDIT) signed a collaboration agreement with a   small cap gene therapy firm called Adverum Biotechnologies (ADVM). The collaboration aims at exploring the delivery of Editas’ genome editing approach with Adverum’s next-generation adeno-associated viral (AAV) vectors to treat up to five inherited retinal diseases that have yet to find treatments or have poor existing remedies. According …
SPARK Therapeutics History Foretells Its Triumph in Gene Therapy

SPARK Therapeutics History Foretells Its Triumph in Gene Therapy

SPARK Spark (NASDAQ: ONCE) is on its way to get an FDA approval of its first gene therapy for a rare genetic condition that causes blindness in children. The firm’s gene therapy product,SPK-RPE65 for inherited retinal dystrophies is designed to be given only once to produce the expected cure. In recent news, Spark is financing through a secondary public offering. The firm plans to use …
Of the Biotech Sector Selloff. Spark Presents its Current and Future Programs

Of the Biotech Sector Selloff. Spark Presents its Current and Future Programs

Of the Biotech Sector’s Selloff Testing the lows in biotech firms’ stock prices is nothing but testing the biotech investors’ degree of vulnerability to fear. Fear leads many biotech investors to hysterically sell their stocks just by hearing negative stories about the sector. The stock selloff continues even when the frightening fabricated stories happen not to make sense that convince mature people to believe them. …
Prohost Letter #388

Prohost Letter #388

Prohost Letter #388 TOPICS - 1. AN INDISPENSABLE SHORT INTRODUCTION 2. GENE EDITING 3. GENE THERAPY 4. CANCER IMMUNOTHERAPY 1. GENE EDITING Exon Skipping: Biomarin (BMRN); Sarepta (SRPT); These firms are waiting FDA approvals for Duchenne Muscular Dystrophy (DMD) Gene Editing Technology Competitors: Cellectis S.A. (CLLS); CRISPR; PTC Therapeutics (PTCT) Firms Using Different Approaches For DMD Treatment (see Below) Zink Finger Tech: Sangamo (SGMO); RNAi …
Prohost Letter #385

Prohost Letter #385

Prohost Letter #385 An Evaluation of Prohost Picks and Some Other Interesting Biotechnology Companies The Top-Tier Group The indiscriminate attack on the biotech sector impacted the best biotech firms more than it did the worst. In other words, the attack devastated the undervalued, without correcting the stock price of overvalued, or even valueless, firms. The problem is that those who instigate computer programs selling of …
Prohost Letter #384

Prohost Letter #384

Prohost Letter #384 The Assassination of the Biotech Sector The Market (Part 2) On Friday September 18, 2015, the biotechnology sector occupied the news headlines for two different reasons. The first was the sudden unexplicable and unexpected plummeting of the biotech sector. The stocks were flying sky high until modday and suddenly, all the biotech stocks, without exception, took a dive. The second reason was the triumph of …