CRISPR Gene Editing Beyond Bringing Cures to disease-causing genes, CRISPR gene editing technology can change the way diseases are being treated and treat diseases that have yet to find a treatment. CRISPR gene editing can play a key role in solving a lot of problems that prevent breakthrough treatments and approaches from reaching their ultimate goals. A best example is chimeric antigen receptor (CAR T) Cell immunotherapy approach to …

Viking Therapeutics Announces Positive Top-Line Results from Phase 2 Study of VK2809 in Patients with Non-Alcoholic Fatty Liver Disease (NAFLD) and Elevated LDL-Cholesterol – The Study Achieves Primary Endpoint, Demonstrating Statistically Significant Reductions in LDL-C in Patients Receiving VK2809  – 57% to 60% Median Liver Fat Reduction Observed in VK2809-Treated Patients  – 77% to 91% of VK2809-Treated Patients Experienced ≥ 30% Reduction in Liver Fat …

Phase 2 clinical trial data of argenx’s (ARGX) product Efgartigimod (ARGX-113) in adult patients with primary immune thrombocytopenia (ITP)  showed a favorable safety and tolerability profile. The results also demonstrated that efgartigimod caused meaningful platelet count improvements across doses and ITP patient classifications, including newly diagnosed, persistent and chronic, and correlated with a consistent reduction in IgG levels. Commenting on the news, Nicolas Leupin, Chief Medical …

Today, we see are observing an increased interest in AcelRx Pharmaceuticals (ACRX). The increased investors’ activity happened following the announcement that an Advisory Committee meeting will soon review the firm’s New Drug Application (NDA) for Dsuvia (Sufentanil) developed for the management of moderate-to-severe acute pain in medically supervised settings in adult patients. Sufentanil is an opioid analgesic currently marketed for intravenous (IV) and epidural anesthesia …

Gilead Sciences (GILD) has news that we felt good about it for a company that saved millions of lives from the minute, but monster viruses HIV and HCV. Curing millions of patients with HCV, has led Gilead to decide that the time has come for bringing cures to many horrible diseases. Yesterday, September 12, the news announced that Gilead and Precision BioSciences have entered into …

Playing buy and sell games with the CRISPR gene editing stocks is sometimes based on scientific articles that meant to caution about possible problems or by patents that were given to some Universities and denied it to others and vice versa. Different Universities back different CRISPR gene-editing firms, which constituted a reason for the volatility of the gene-editing stocks. Finally, CRISPR Therapeutics (CRSP), Intellia Therapeutics …

In the Week in Review posted yesterday on our website that is experiencing several attempts on its life, we wrote about some firms, including Amgen. In the Amgen’s article we tried to let the reader recognize the importance of Amgen’s (AMGN) asthma product tezepelumab and how it differs from all other asthma drugs on the market, including the conventional tablets, inhalers and infusions. The Novel …

The Week in Review #29 PROHOST AGGRESSIVE PORTFOLIO  - The aggressive portfolio hosts biotech firms with solid scientific fundamentals and prominent scientists whose stocks have plummeted following some setbacks. A setback could be a failure of an investigational product to meet the endpoints of its clinical trial or that the small biotech firm has taken long to achieve its goals . . . This content …

Gilead Sciences’ (GILD) and Galapagos’ (GLPG) product filgotinib – a highly selective JAK1 inhibitor has met the primary efficacy endpoint in Phase 2 TORTUGA trial in adults with moderately to severely active ankylosing spondylitis (AS). Patients treated with filgotinib achieved significantly greater improvements compared to those who took a placebo. The results came from the Phase 2 of the TORTUGA – a multi-center, randomized, double-blind, …

The disease, mucopolysaccharidosis (MPS) type II, also known as the horrible Hunter syndrome is a killer with no specific treatment anywhere, except enzyme replacement therapy (ERT) that is not stopping the complications of the disease or preventing death. Sangamo (SGMO) made a presentation at the Society for the Study of Inborn Errors of Metabolism in Athens, Greece to demonstrate preliminary results, which included the safety …

The Week in Review #28 The approval of revolutionary products created by biotech firms with solid scientific fundamentals, superior scientists and advanced technologies is what motivates us to pick for investment biotech and biopharmaceutical firms. So, let’s see what has been approved in the parting week  from firms that we picked for investment.       IMPORTANT APPROVALS       Prohost Picked Firm     …

We reiterate that undeniably outstanding news about gene therapy is taking time to penetrate investors’ ears. They are still falling victim to bad rules of thumb and critics’ magnifications of the negative impact of some stocks’ dilution when companies with superior programs try to finance through a public offering. The stocks’ punishments following financing is not a rule to apply to all firms, especially those …

A complete response letter (CRL) sent by The Division of Metabolism and Endocrinology Products of the United States’ FDA to Ionis Pharmaceuticals (IONS) and its affiliate Akcea Therapeutics (AKCA) regarding Waylivra™ (volanesorsen) carried like usual bad news. The FDA has decided against approving Waylivra, the drug designed to treat the lipid disorder known as familial chylomicronemia syndrome (FCS). Familial chylomicronemia syndrome is an ultra-rare, devastating hereditary …

Before we sell PTC Therapeutics (PTCT), after the stock had reached and surpassed our latest and highest 2018 target, the firm did the right thing that we expected was good enough to encourage investment in this firm.  The good thing is acquiring Agilis Biotherapeutics – a small firm that succeeded in creating innovative gene therapy programs for rare genetic disorders that affect the central nervous …

Portola (PTLA) has two important approved breakthrough products and an investigational product having promising results on very difficult-to-treat cancers. Instead of soaring following the approval of its two drugs and the good results coming from the investigational third drug, Portola’s stock took several dives, which raised the eyebrows of many investors and motivated more shareholders to sell PTLA. Let us see whether the firm deserves …

When the Goal is Reaching into the Future, Pessimists Have No Place on the Ship The revolution in the biotech sector has proven to be moving fast towards reaching goals that were impossible to imagine a couple of years ago that they could be attainable. This is not true only regarding lay investors, or desperate patients, but also regarding the most professional clinicians, researchers and …

Celyad (CYAD) is an immuno-oncology firm, which created CAR T CELL treatments for cancers, including  solid tumors. Celyad announced its consolidated financial results for the six-month period ending 30 June 2018. Celyad’s report is endorsed by Europe. Dr. Christian Homsy, Celyad’s CEO started by expressing the firm’s great enthusiasm towards the progress it has made in the first half of 2018, which include a significant advancement of …

WE ARE LIVING AN HISTORIC BIOLOGICAL REVOLUTION. Unfortunately, the undeniably outstanding news has not yet penetrated the ears of many investors. They are still falling victim to the stock market traders’ games and to critics’ and bloggers’ magnifications or fabrications of negative news. Many investors end up selling their thoughtfully chosen highly scientific biotech firms to regret later on what they have done when their …

Vertex Pharmaceuticals (VRTX) announced that the Pharmaceutical Benefits Advisory Committee’s (PBAC) in Australia recommendation the listing of Orkambi® (lumacaftor/ivacaftor) on the Pharmaceutical Benefits Scheme (PBS). The recommendation is for cystic fibrosis people ages six and over who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Many thousands of patients worldwide are already receiving lumacaftor/ivacaftor in countries where it is reimbursed …

The FDA Approves Regeneron’s Product EyLea® (aflibercept) sBLA in Wet Age-Related Macular Degeneration Regeneron (REGN) has two good news. The first is from one of its approved products, EYLEA® announcing that the U.S. Food and Drug Administration(FDA) has approved a supplemental Biologics License Application (sBLA) for EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD). The sBLA was based on second-year data from the …