XENE – Epilepsy and Advocacy Under the New FDA

Xenon Pharmaceuticals (XENE) is a clinical stage biopharmaceutical company focusing on discovering and developing innovative therapeutics for neurological disorders. XENE has an extensive knowledge of human genetics and diseases caused by mutations in ion channels, known as channelopathies, focusing their efforts on novel product pipeline of CNS-central nervous system therapies, to address areas of high […]

FDA GT Draft Guidance is Bullish for Sarepta's DMD Pivotal Trial

Yesterday July 11, 2018 the FDA FDA issues 6 new draft guidance documents for gene therapy, clinical development and manufacturing guidance. Human Gene Therapy for Rare Diseases – FDA Draft Guidance for Industry https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM610802.pdf   The FDA guidance noting the pre-existing antibody to GT, this is the strongest SRPT’s & Dr. Mendell point, choosing the AAVrh74 which […]

PSTI – Ahead of IC Phase-2 Top-Line Results

PLURISTEM (PSTI) a cell therapy company, developer of placenta-based cell therapy product candidates for the treatment of multiple ischemic, inflammatory and hematologic conditions, company I’m following for years.  Pluristem trading under the radar and getting even more interesting these days as they already enter late-stage trials in 3 indications, gained the FDA’s Fast Track Designation, EMA’s […]

SRPT Was and Still The DMD Gene Therapy Leader

Today after the market close, Solid Biosciences (SLDB) announced it has received notification from the U.S. Food and Drug Administration (FDA) that IGNITE DMD, its Phase I/II clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold. The news sent SLDB share price down to $11.90 or […]

Dr. James Wilson Paper About Safety Concerns, Is Not Sarepta’s Concern!

Today morning, most of the gene-therapy stocks including SLDB, AVXS, ONCE, RGNX, BOLD, ABEO and even SRPT Stumbled following Dr. James Wilson paper published online “Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN” (link to the paper) highlighting the toxicity in NHP- non-human primate study. […]

A Quick Technical Look At SRPT Full Year Chart

Sarepta Therapeutics, Inc. (SRPT) Nasdaq   A quick look at Sarepta’s yearly chart, from the spike to $63.73 after Exondys-51 FDA approval and the drop to $26.66 lowest point by Dec 30th, the last trading day of 2016. What is important to watch is the big picture, as we are right now above the Fibo-50% retracement […]

A Deep Dive in SRPT Chart!

Sarepta Therapeutics, Inc. (SRPT) -Nasdaq During the past six weeks we had so many stories, with or against SRPT approval, where a 10-million shares short sellers are still traped in this name with a significant loss after the FDA approval, and they are trying nonstop any way or attack to find a way to make […]

SRPT – YEAR 4 DATA ANALYSIS

  YEAR 4 DATA ANALYSIS Review of Eteplirsen to treat DMD By: @boscaptn, et al.   Compiled From SAREPTA Public Data For Week 216 (Year 4) of Study 201/202 for Exon 51   Disclaimer: NO FINANCIAL ADVICE – The Information in this report is provided for education and informational purposes only, without any express or […]