Recent Gene Therapy News On August 20, 2020 Audentes Therapeutics, which was taken over by Astellas (ALPMY) for over $3 billion, announced that a third patient has passed away in a clinical trial evaluating its gene therapy product AT132 for the treatment of the life-threatening neuromuscular X-linked Myotubular Myopathy (XLMTM). The neuromuscular disease is characterized by extreme muscle weakness, respiratory failure and early death. Preliminary …

Regenxbio Positive Data for RGX-314 Regenxbio (RGNX) reported positive one-year data from patients in Cohorts 4 and 5 from Phase I/IIa trial of RGX-314 for wet age-related macular degeneration (wet AMD). Regenxbio plans to initiate a pivotal program for the subretinal delivery of the gene therapy RGX-314 in patients . . . This content is for paid subscribers. Please click here to subscribe or here to log …

Beyond the firms that are speeding towards bringing prophylactic vaccines against the COVID-19 infection into the clinic we have to consider the other firms that have been negatively affected by the virus pandemic; we expect them to rebound and surge when the virus loses its ability to kill. We decided to start with gene therapy companies that we believe have suffered from the delays of …

Exelixis, Takeda and Japanese RCC Patients Today we learned from Exelixis (EXEL) that Takeda Pharmaceutical Company Limited, which is responsible for the clinical development and commercialization of Exelixis' oncology drug  CABOMETYX® (cabozantinib . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Regenxbio, Moderna and Others Regenxbio (RGNX) announced several poster presentations including interim data from its Phase I/II trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II

Regenxbio Results from RGX-121 for MPSII Regenxbio (RGNX) announced interim data from Phase I/II trial of RGX-121 for Mucopolysaccharidosis Type II (MPS II). RGX-121 is designed to deliver the gene that encodes iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) using the AAV9 vector. About Mucopolysaccharidosis Type . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The Market May be Plummeting But Prohost Picks are Not Biotech stocks had a strong start, but the gains disappeared in many stocks as the market demonstrated bearishness following a long-lasting rally. Currently, during the strong economic conditions, commentators attribute the reasons for the market’s upside and downside moves to China’s positive, or negative ongoing negotiations rather than to fair correction of overbought or oversold …

Regenxbio Misleading Selloff A selloff of Regenxbio (RGNX) stock, following the FDA’s partial hold on the intrathecal administration of AVXS-101, is unwarranted and totally misleading.   Why? Because the selloff of RGNX gave the impression that the gene therapy Zolgensma, using the adeno-associated virus (AAV) vector , developed by Regenxbio, has failed, which is not true. The partial hold was put only on the product …

RegenxBio Rebounding RegenxBio (RGNX) has the viral vectors which could safely deliver genes into the cells. The firm has improved the adeno-associated viruses making them safe and effective gene delivery vehicles. One of the three currently approved gene therapy products, Zolgensma® for pediatric spinal muscular atrophy (SMA), marketed by Novartis, used RegenxBio’s adeno-associated . . . This content is for paid subscribers. Please click here to …

RegenxBio Interim Data from Phase I/IIa Trial of RGX-314 for Wet AMD RegenxBio (RGNX) announced interim data from the ongoing Phase I/IIa trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). Here are the results as presented by the Company. The results were presented by Jeffrey S. Heier, M.D., Co-President and Director . . . This content is for paid subscribers. Please …

Prohost Letter #436 What Are We Experiencing Now? The bizarre market underperformance progressing rather than regressing and the good economy and the employment gains are the least utilized factors after being the most influential on the stock market performance. We are witnessing the most bullish analysts being confused, hesitating to give their followers a crystal clear explanation about the market’s skepticism, anger and volatility. The …

Migraine's Really Effective Treatments The years 2018 and 2019 could be called the years of migraine’s really effective treatments. Three Calcitonin-gene-related peptide (CGRP) monoclonal antibody drugs have been approved in 2018 and 2019. They are: Aimovig for Amgen (AMGN . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Diseases of the Back of the Eye Diseases of the back of the eye, such as: age-related wet macular degeneration, diabetic retinopathy and diabetic macular edema, are currently managed with injections of anti-vascular endothelial growth factor (anti-VEGF) drugs into the gel-like substance (the vitreous humor) that fills the eye between the lens and the retina. The currently used age-related wet macular degeneration injections are: Lucentis, which belongs …

RegenxBio License Agreement with Pfizer RegenxBio (RGNX) announced a license agreement with Pfizer (PFE). Under the terms of the agreement RegenxBio has granted Pfizer a non-exclusive worldwide license, with rights to sublicense, to RegenxBio’s NAV AAV9 vector for the development and commercialization of gene therapies for Friedreich’s ataxia (FA). The most common hereditary ataxia (neurological lack of muscle coordination). RegenxBio will receive an upfront payment and has …

Small Biotech Firms That Can Become Top-Tier Biotech Giants We Start With... Abeona Therapeutics Abeona Therapeutics (ABEO) is a clinical-stage biopharmaceutical gene and cell therapy company. In September 2016 Abeona acquired an exclusive worldwide license of a next-generation gene therapy AAV capsid portfolio from University of North Carolina at Chapel Hill.  The AIM™ vector system . . . This content is for paid subscribers. Please click here to subscribe …

Zolgensma Findings 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 Novartis (NVS) presented interim results for 22 babies with Type 1 SMA which is considered the . . . This content is for paid subscribers. Please click here to subscribe …

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 …

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 …