Intellia Therapeutics Granted Orphan Drug Designation for NTLA-2001 Intellia Therapeutics (NTLA) announced that the U.S. FDA has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis. This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body. NTLA-2001 has the potential to be the first single-dose treatment for ATTR amyloidosis as it …

Rocket Pharmaceuticals Inc Announces Successful Results at the ESGCT Gene therapy and gene editing plus other gene and genome-based therapeutics are the only treatments expected to lead to  miraculous cures. While genetic manipulation for the treatment of severe life-threatening diseases is still moving towards perfection, gene therapy and gene editing are accomplishing miraculous long-term effective treatments, saving thousands of lives where there are no currently …

Intellia Therapeutics and SparingVision Collaboration Intellia Therapeutics (NTLA) and SparingVision - a genomic medicine company developing vision saving treatments for ocular diseases, announced a strategic collaboration to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases. Intellia Therapeutics is to grant SparingVision exclusive rights to Intellia . . . This content is for paid subscribers. Please click here to subscribe or here to …

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 …

Denali Therapeutics News Denali Therapeutics (DNLI) -  a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases.  Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the BBB and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San . . . This content is for …

Two Clinical-Stage Firms Rallied Yesterday On a day when the Stock Market was crushed, taking down with it shareholders’ money for no reason other than the self-serving untouchable agendas, two small biotechnology companies staged rallies. Atea Pharmaceuticals and Xenon Pharmaceuticals deserved the boosting of their stocks.   Atea Pharmaceuticals Atea Pharmaceuticals (AVIR) - a clinical-stage biopharmaceutical company, reported for the first time . . . …

Merck and Ridgeback Biotherapeutics COVID-19 News  Merck (MRK) and Ridgeback Biotherapeutics today announced that molnupiravir (MK-4482, EIDD-2801) - an investigational oral antiviral medicine, has significantly reduced the risk of hospitalization or death at a planned interim analysis of the Phase 3 MOVe-OUT trial in at risk, non-hospitalized . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Valneva SE and Pfizer Inc Announced More Positive Trial Results of VLA15 for Lyme Disease What is Lyme Disease? What is the vaccine? What is the importance of the vaccine and the company that created it?    Lyme disease is an infection caused by Borrelia burgdorferi bacteria transmitted to humans by infected Ixodes ticks. Lyme disease is considered the most common vector- borne illness in the Northern Hemisphere. The U.S. …

Regeneron Welcomes the World Health Organization's Recommendation for Use of REGEN- COVTM to Treat COVID-19 The World Health Organization (WHO) updated its Therapeutics and COVID-19: living guideline to include the casirivimab and imdevimab monoclonal antibody cocktail, known as REGEN-COVTM in the U.S. and Ronapreve® in other countries. The guideline conditionally recommends REGEN-COV to treat high-risk non-hospitalized patients with non-severe COVID-19 and seronegative (no measurable . …

Gilead Sciences New Data from Trodelvy® Study Gilead Sciences (GILD) has recently announced new data from the Phase 3 ASCENT study evaluating Trodelvy® in patients with relapsed or refractory metastatic triple-negative breast cancer (TNBC) who received two or more prior systemic therapies, at least one of them for metastatic disease. Trodelvy has shown to improve progression-free survival (PFS . . . This content is for paid …

The most important news in biological sciences is the high degree of success the biological revolution can reach. We can feel the great value every time an untreatable, devastating condition is successfully treated thanks to discoveries made possible through the information brought in by the unparalleled revolution that allows for a better understanding of the living body mechanisms of actions in health and in diseases. …

Cara Therapeutics and Vifor Pharma Granted US FDA Approval of KORSUVA™ Indeed, the U.S. Food and Drug Administration (FDA) approved Cara Therapeutics (CARA) and Vifor Pharma injectable product KORSUVA™ (difelikefalin) - a first-in-class kappa opioid receptor (KOR) agonist that targets the body’s peripheral nervous system, for moderate-to-severe pruritus associated with chronic kidney . . . This content is for paid subscribers. Please click here to subscribe or here to …

Travere Therapeutics Sparsentan for IgAN Positive Results Phase 3 PROTECT Study of Travere Therapeutics (TVTX) product sparsentan for the treatment of IgA nephropathy (IgAN) demonstrated positive topline interim results. The PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance, demonstrating a greater than a threefold reduction of proteinuria from baseline after 36 weeks of treatment . . . This content is for paid …

Akero Therapeutics Inc Akero Therapeutics Inc (AKRO) is a clinical-stage cardio-metabolic company developing transformational treatments for non-alcoholic steatohepatitis (NASH), a disease without any approved therapies. Akero’s investigational drug product candidate, efruxifermin (EFX), is a differentiated Fc-FGF21 fusion protein engineered to mimic the balanced biological activity profile of native FGF21, an endogenous hormone that alleviates . . . This content is for paid subscribers. Please click here …

Vir Biotechnology: What We've Learned from Dr. George Scangos George Scangos, PhD. is the chief executive officer of Vir Biotechnology (VIR), we don't know if you remember him from our previous postings, so we will remind that Dr. Scangos has said that Vir Biotechnology made strong progress across its extensive infectious disease portfolio, the most notable of which . . . This content is for …

Reata Pharmaceuticals Two Stories  Omaveloxolone in Patients with Friedreich’s Ataxia  Several communications between Reata Pharmaceuticals (RETA) and the FDA have taken place in the past regarding omaveloxolone for Friedreich’s Ataxia (FA) without reaching clear results that could satisfy either the Firm or the FDA. However, recent communication by the FDA seems to have finally satisfied both.  Following . . . This content is for paid …

Novartis Partial Clinical Trial Hold for OAV-101 IT for SMA Lifted Novartis (NVS) announced that the U.S. FDA has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. The decision to lift the hold was based on data from . . . This content is for paid subscribers. …

Cassava Sciences There is no doubt in our minds that Cassava Sciences’ (SAVA) product simufilam will play a major role in the treatment of Alzheimer’s disease. We have no doubt that simufilam has improved cognition in patients with mild to moderate Alzheimer’s disease. The cognition gained 3.0 Points on ADAS-Cog in 9 months. The Alzheimer’s patients’ disordered . . . This content is for paid …

Cassava Sciences Inc Following Cassava Sciences Inc successful and promising presentations, all the titles of the news and articles written by fair professionals have been as follows: “Cassava Sciences announces positive biomarkers’ data and positive Cognition Data following the treatment with Simufilam” These titles never moved from the Internet even after a huge sell-off occurred.  Indeed, the improvement in cognition demonstrated by the data was …

Cassava Sciences Soared Cassava Sciences (SAVA) stock soared today. The firm announced that it will present two new clinical datasets for its product candidates at the 2021 Alzheimer’s Association International Conference (AAIC), which will be held virtually in Denver, CO from July 26th through July 30th. Cassava Sciences’ investigational product candidates include simufilam treatment . . . This content is for paid subscribers. Please click here to subscribe or …