Novartis Gene Therapy Zolgensma Will Be Approved. The FDA Approves a Merck Combination for First-Line Renal Cell Carcinoma. RegenxBio and Exelixis Are in Great Shape.   Novartis' Gene Therpay Product Zolgensma The FDA submission of the gene therapy product Zolgensma was based on positive findings from a trial of 15 babies treated with it. Recently; however, at the Orlando meeting of the Muscular Dystrophy Association …

The Week in Review #39 During the Departed Week Three Prohost Portfolio Picks Hit Their 52-Week Highs The three picks referred to in the above title are, Novartis (NVS), Array (AARY) and Ionis (IONS). Previous Prohost Letters discussed Novartis and Array and continued to remind that they are Prohost favorites. The time has come to take a deeper look at Ionis; starting with the reasons …

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. …

RegenxBio (RGNX), a clinical-stage gene therapy firm with a proprietary NAV® Technology Platform, will host an analyst and investor event focused on its ophthalmology program; including its lead product candidate RGX-314, on Thursday, February 21, 2019, from 9:00 a.m. to 12:00 p.m. EST in New York City. A live webcast will be available on the company’s website. The agenda will include discussion on RegenxBio’s NAV® Technology Platform, a review of the market landscape and unmet need …

The United States FDA granted AMT-061 Breakthrough Therapy Designation and 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 …

FDA Granted Rare Pediatric Disease Designation for RegenxBio’s Product RGX-181 for CLN2 RegenxBio RegenxBio (RGNX), a development-stage firm that focuses on designing and developing gene therapy based on its proprietary NAV® Technology Platform, announced that the U.S. FDA granted Rare Pediatric Disease Designation to its RGX-181 therapy. RegenxBio is a leading clinical-stage biotechnology company that promises to cure genetic-derived diseases with its gene therapy. RegenxBio’s NAV Technology Platform is …

Now You See it, Now You Don’t Deliberate Omissions of Real Facts Real facts might be displayed or concealed depending on the news announcers’ or bloggers’ self-interests. The following contains cases of deliberate or unintentional omission of facts that led to the decline of fundamentally sound biotech and biopharmaceutical firms in bullish market during the departed trading week . . . This content is for paid subscribers. Please …

The Week in Review #35 Politics and the Market The market tumbled today as Theresa May, the Prime Minister of England, delayed the Brexit vote!  The question becomes, what will be put forth next as a legitimate reason for bringing the market down and eroding investors’ gains? The weather? A war among gangs in a fourth-world country? Maybe the resignation of the chairman of a …

Today, the market took another beating as a result of either an intentional or an unintentional lack of clearance by professionals in describing correctly the standing of employment and other current economic realities. They pinpointed irrelevant reasons in an effort to justify the Stock Market's tumbling.  One big headline was that 150,000 added jobs did not meet expectations. They never explained whether those jobs were all …

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 …

We hope you all took advantage of the Thanksgiving holiday to rest and relax. We did. In Today’s NEWS RegenxBio Offers Rocket Pharmaceuticals exclusive, worldwide, rights to adenoviral vector NAV AAV9 for the development of treatments for Danon Disease RegenxBio (RGNX) announced that it awarded an exclusive, worldwide, license agreement to Rocket Pharmaceuticals (RCKT) so it can develop gene therapy for Danon disease. The exclusive license …

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 …

RegenxBio (RGNX) has granted Abeona Therapeutics (ABEO) an exclusive worldwide license to its NAV AAV9 viral vector (subject to certain non-exclusive rights previously granted for MPS IIIA) for the treatment of four diseases: Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB), Infantile Batten Disease, also known as neuronal ceroid lipofuscinosis type 1 (CLN1 Disease) and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 …

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 …

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 …

The Week in Review #29 PROHOST AGGRESSIVE PORTFOLIO  - The aggressive portfolio hosts biotech firms with solid scientific fundamentals and prominent scientists whose stocks have plummeted following some setbacks. A setback could be a failure of an investigational product to meet the endpoints of its clinical trial or that the small biotech firm has taken long to achieve its goals . . . This content …

The Week in Review #28 The approval of revolutionary products created by biotech firms with solid scientific fundamentals, superior scientists and advanced technologies is what motivates us to pick for investment biotech and biopharmaceutical firms. So, let’s see what has been approved in the parting week  from firms that we picked for investment.       IMPORTANT APPROVALS       Prohost Picked Firm     …

We reiterate that undeniably outstanding news about gene therapy is taking time to penetrate investors’ ears. They are still falling victim to bad rules of thumb and critics’ magnifications of the negative impact of some stocks’ dilution when companies with superior programs try to finance through a public offering. The stocks’ punishments following financing is not a rule to apply to all firms, especially those …

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 …

Regeneron Pharmaceuticals (REGN) and bluebird bio, Inc. (BLUE) announced a collaboration to apply their respective technology platforms to the discovery, development, and commercialization of novel immune cell therapies for cancer. The collaborators will specifically leverage Regeneron’s VelociSuite® platform technologies for the discovery of human antibodies as well as T cell receptors (TCRs) directed against tumor-specific proteins and peptides, and bluebird bio will contribute its field-leading expertise in gene transfer and …