Regenxbio Positive Results Regenxbio (RGNX) announced positive results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for patients with Mucopolysaccharidosis Type 2 (MPS II), also known as Hunter syndrome. The results were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024. The totality of evidence from the CAMPSIITE trial continues to . . . This content is for …
RegenxBio Today RegenxBio (RGNX) announced it completed a successful Pre-Biologics License Application (BLA) meeting with the FDA for the RGX-121 treatment of Mucopolysaccharidosis Type II (MPS II). The FDA continues to be aligned with RegenxBio's plan to use cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker of brain disease activity, as a . . . This content is for paid subscribers. …
Regenxbio Product RGX-121 Achieves Primary Endpoint From Regenxbio (RGNX) we learned that results from the Phase I/II/III CAMPSIITE® trial of the firm’s product RGX-121 for the treatment of 5 years old patients diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, demonstrated that the pivotal phase of the trial met its primary . . . This content is for paid subscribers. Please …
Regenxbio in the NEWS Today, the U.S. FDA granted Fast Track designation for Regenxbio (RGNX) Gene therapy RGX-202 a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne). Therapies granted Fast Track designation are given the opportunity for more frequent interactions with the FDA and may qualify for priority review. The FDA has granted RGX-202 . . . This content is for paid …
Prohost's Beliefs About the Status of the Market That the market is still volatile and Bear. Investors in the biotech sector who intend to bet on clinical stage firms must restrict their choices to companies that already have proof of concept regarding safety and efficacy in some midterm and pre-term clinical trial results. Caution is still important for clinical-stage firms’ stocks that have encouraging clinical …
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 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 …
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 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 …
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 …
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. …
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 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 …
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 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 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 …
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 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 …
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