Tag: Regenxbio (RGNX)

Important Data Will Be Presented by Regenxbio on August 30th

Regenxbio to Present Important Data  Regenxbio (RGNX) will present important material at the Society for the Study of Inborn Errors of Metabolism Annual Symposium, taking place in Germany from August 30 through September 2, 2022. The presentations are meant to highlight new data from the Phase I/II/III CAMPSIITE™ trial of RGX-121, an investigational one-time AAV Therapeutic . . . This content is for paid subscribers. …

Regenxbio to File a BLA Using the Accelerated Approval Pathway for RGX-121 for the Hunter Syndrome

Regenxbio in the NEWS  Regenxbio (RGNX) announced, on August 3, 2022, its intention to file a Biologics License Application (BLA) in 2024 using the FDA's accelerated approval pathway for RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome.   The Company has also announced that a pivotal program for RGX-121 is . . . This content is for paid …

Shorting Biotech Firms with Solid Fundamentals

Prohost Comprehensive Letter #451 Shorting Biotech Firms with Solid Fundamentals Unfair shorting of promising stocks seems to have become the norm during the harsh circumstances that not only the United States but several other countries have been living under for more than two years. The bad circumstances in the United States seem to be motivating unfair short-sellers to take advantage of bad news and cause …

Comprehensive Prohost Letter #449 – Reality

Comprehensive Prohost Letter Issues Reality The Best of words in informative texts are the smallest in number and the most truthful in contents. That’s what Prohost believes, especially in the presence of the cruel circumstances our entire planet is currently living. Last Friday, the three indexes of the stock market had their best performances since the 4th quarter of 2020. Why? We don’t know, but …

What Happened Over the Past Two Days and Biotech

Some Gains in Biotech Stocks During the War in Europe On Tuesday, Mar 8,2022 the United States’ government decided to ban the importation of Russian oil, liquefied natural gas and coal. This decision was made to further deprive Russia from the resources that enable it to continue with the unwarranted war against Ukraine. This resolution has come at a time when the price of oil …

An Important Milestone for Regenxbio Treatment of Wet AMD

Regenxbio Good News for wet AMD On January 10, 2022,  Regenxbio Inc. (RGNX) announced the initiation of ASCENT, the second of two Phase 3 pivotal trials to evaluate the efficacy and safety of subretinal delivery of RGX-314 in patients with wet age-related macular degeneration (wet AMD). RGX-314 is being investigated as a potential one-time gene therapy for . . . This content is for paid subscribers. …

The FDA Granted Orphan Drug Designation to Regenxbio Investigational Gene Therapy Drug RGX-202 for Duchenne Muscular Dystrophy

Regenxbio Granted Orphan Drug Designation for RGX-202 Regenxbio (RGNX) announced the U.S. FDA granted Orphan Drug Designation for RGX-202 - a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy. RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with a unique C-terminal domain and a . . . This content is for paid subscribers. Please click here to subscribe or here to log …

Regenxbio Good News About RGX-314 for Diabetic Retinopathy

Regenxbio Announced Data from Phase 2 ALTITUDE Trial of RGX-314 for DR Regenxbio (RGNX) announced initial data from the ongoing Phase 2 ALTITUDE™ trial of RGX-314 for the treatment of diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME) using in-office suprachoroidal delivery. Regenxbio is a clinical-stage biotechnology company seeking to improve lives . . . This content is for paid subscribers. Please click here to …

Comprehensive Prohost Letter Issues

The Comprehensive Prohost Letter Issues The Comprehensive Prohost Letter Issues will constitute a chain of 7 issues. Today’s issue will include the following: Pinpointing and selecting the biotech firms that demonstrate readiness to treat untreatable, devastating diseases. Presenting an important antibody cocktail for the treatment of COVID-19. Stating our position regarding the Alzheimer’s disease firm Cassava Sciences. Pinpointing Prohost’s . . . This content is …

Roche and Spark Therapeutics: Gene Therapy SPK-8016 Demonstrating Promising Results on Adults with Severe Hemophilia A

Roche Group and Spark Therapeutics Gene Therapy Showing Promising Results Looking for gene therapy treatments for chronic debilitating life-threatening diseases we fell on news from Spark Therapeutics, a member of the Roche Group (RHHBY); the firm announced preliminary data from part one of the ongoing Phase 1/2 open-label, non-randomized, dose-finding study of the investigational SPK-8016 . . . This content is for paid subscribers. Please click here …

Regenxbio: Advancing its Gene Therapy Pipeline and its Rare Neurodegenerative Disease Platform with Non-Dilutive Capital

Regenxbio Regenxbio (RGNX) announced that it is selling a portion of its royalty rights from the net sales of Zolgensma® by Novartis to entities managed by Healthcare Royalty Management, LLC (HCR) for $200 million. This transaction provides immediate, non-dilutive capital to Regenxbio in order continue the development of its gene therapy pipeline products and complete its internal manufacturing capabilities. Morgan Stanley & Co. LLC served …

The Biotechnology Industry

Prohost Letter #443 The Biotechnology Industry The Biotechnology Industry revolutionized the arts of life sciences with accomplishments promising a tremendous improvement in the treatment of chronic, debilitating, progressive and life-threatening diseases. The improved approaches have been game changers in the treatment of cancers and genetic-derived hereditary and non-hereditary chronic diseases. The out-performance was made possible through the biotech industry’s creativity, innovation and remarkable productivity. The …

Regenxbio Announced the First Patient has been Dosed in the Phase I/II trial of RGX-111 for MPS I

Regenxbio Dosed First Patient with RGX-111 for MPS I Regenxbio (RGNX) has dosed the first patient with RGX-111 in the Phase I/II trial for the treatment of Mucopolysaccharidosis Type I (MPS I). About Regenxbio Product RGX-111 RGX-111 is an investigational one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS) using the NAV AAV9 vector. RGX-111 is a …

What We Expect to Hear at the Regenxbio Conference Call Today

Regenxbio Conference Call Information Regenxbio will host a conference call today, at 4:30 pm est today, November 4, 2020, to discuss its financial results for the quarter, ended September 30, 2020, and recent operational highlights. To access the live call by phone dial (855) 422-8964 (domestic) or (210) 229-8819 (international) and enter the passcode 4107819. To access a live or recorded webcast of the call …

Why We Expect a Lot from the Following Biotech Firms

Compugen Compugen (CGEN) announced the presentation of new research data that further support PVRIG as a potentially promising target for cancer immunotherapy. These data suggest that PVRIG inhibition may enhance T cell priming and infiltration into tumors and provide further evidence supporting the potential advantages of targeting PVRIG alone and in combination with TIGIT and PD-1 inhibitors, in tumors . . . This content is for …

SARS-CoV-2 Virus vs The President of the United States

President Trump, the First Lady and COVID-19 Indeed, the complicated, nasty SARS-CoV-2 virus managed to infect President Trump and the First Lady when we were expecting a vaccine to soon be ready to protect the country and the world from COVID-19. The president’s physicians did not treat the him with any of the investigational vaccines in late-phase trials; vaccines being developed by: Moderna (

What’s Going on with Gene Therapy?

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 . . . This content is for paid subscribers. Please click here …

Regenxbio: Positive One-Year Data from the Firm’s Gene Therapy RGX-314 for Wet AMD

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 …