Sangamo Therapeutics Preliminary Results Evaluating Isaralgagene Civaparvovec for Fabry Disease Sangamo Therapeutics (SGMO) announced preliminary results from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920 - a wholly owned gene therapy product candidate, for Fabry disease. As of the September 17, 2021 cutoff date, results from the four patients treated in the first two dose cohorts (0.5e13 vg . . . This content is for …

bluebird bio Suspends LentiGlobin Studies bluebird bio (BLUE) placed its Phase 1/2 (HGB-206) and Phase 3 (HGB-210) studies of LentiGlobin gene therapy for sickle cell disease (SCD), bb1111, on a temporary suspension due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Gilead’s CAR-T Product Yescarta Results Kite, a Gilead (GILD) subsidiary, announced new data from the ZUMA-1 trial of the CAR T Yescarta (axicabtagene ciloleucel) in adult patients with refractory large B-cell lymphoma. The data from the pivotal phase 2 study demonstrate that patients with refractory large B cell lymphoma were alive three years following a single infusion of Gilead’s CAR T immuno-oncology product Yescarta.  Analysis from …

The Market May be Plummeting But Prohost Picks are Not Biotech stocks had a strong start, but the gains disappeared in many stocks as the market demonstrated bearishness following a long-lasting rally. Currently, during the strong economic conditions, commentators attribute the reasons for the market’s upside and downside moves to China’s positive, or negative ongoing negotiations rather than to fair correction of overbought or oversold …

Sangamo and Pfizer Gene Therapy SB-525 Resulted in a Sustained Increase in Factor VIII Levels in Hemophilia Patients Sangamo Therapeutics (SGMO) and Pfizer (

Sangamo’s Stock Price Soars Sangamo Therapeutics’ (SGMO) stock soared more than 52% in pre-market trading following the announcement with Pfizer (PFE), of interim data from Phase 1/2 Alta study evaluating the firm’s gene therapy product SB-525 for severe hemophilia A.  The Product, SB-525 SB-525 comprises a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. The …

Prohost Letter #427 End of the Year Decisions ~ Part 2 As a result of the unexpected volatility in the stock market, we carefully reassessed each and very stock we picked for the Prohost Portfolio. Consequently, we had to change many of our 2019 targets in all 7 tables which represents the whole portfolio of companies we believe are offering lucrative investment opportunities. On January 3, …

We have a few small clinical stage firms in the Prohost Portfolio that were established with the goal of designing safer and more effective versions of the very promising CAR T immuno-oncology products. We intend to revisit these firms and see how far they have reached on what can be described as a very hard road towards the realization of these small ambitious firms’ goals. Among …

Prohost Letter #425 When Common Sense Fails to Explain the Market Performance It is not unusual for the stock market to be in a selloff mood for a few days. However, in the last couple of months, the market unexpectedly plunged deep into the ocean of the unknown. The exaggerated volatility was nerve-wracking for professional analysts. This is especially true for candid analysts who base …

Prohost Letter #424 Following the Stars Less Science and More Logic We reiterate that while a human star is born every once in a while on Earth, several stars born daily in research labs may be designated every other day by the FDA and other health regulatory agencies around the world. These agencies call these potential stars’ breakthrough drugs, but not all the molecules designated …

The Week in Review #31 IN THE DEPARTED WEEK NOVEL MIGRAINE DRUGS ARE APPROVED ELI LILLY AFTER AMGEN AND TEVA In less than five months, the FDA approved three products belonging to four drug developers – all targeting calcitonin gene-related peptide (CGRP) for the prevention of migraine headache. The latest for Eli Lilly (LLY) last week . . . This content is for paid subscribers. …

The Week in Review #30 FROM THE DEPARTED WEEK In the past week, we came across a lot of news announcing research, clinical trial results, collaborations, product approvals and acquisitions. Much of the departed week’s news though, does not represent complete stories, especially the ones related to research. They represented early works in progress offering no concrete results that we can evaluate. Important news for …

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 …

ProhostLetter #423 TOMORROW’S THERAPEUTICS TODAY For a more or less fair evaluation of biotechnology and pharmaceutical firms, analysts currently rely for their assessment on criteria that include quarterly financial results, products’ sales revenues, and year over year growth of each and every marketed therapeutic. When it comes to clinical-stage firms, the logic implies that these criteria cannot be relied on for evaluation, as most of …

The Week in Review #26 IN THE BIOTECH WORLD - RESEARCH - IMPROVING CRISPR GENE EDITING The problem cited with gene editing is that the editing may stay switched on after it succeeded in reaching its goals. To prevent further unwarranted mutations, scientists from the Bath and Cardiff University have invented a switch that aims at controlling protein expression in cultured cells and mouse embryos with the …

SPARK THERAPEUTICS, SANGAMO THERAPEUTICS AND SALK INSTITUTE CURING HEMOPHILIA?   Hemophilia is an inherited bleeding disorder that causes abnormal or exaggerated bleeding and poor blood clotting. Hemophilia A and Hemophilia B are inherited in an X-linked recessive genetic pattern, so males are commonly affected while females are usually carriers of the disease. Hemophilia A is caused by clotting Factor VIII deficiency and hemophilia B (Christmas disease) is caused by Factor IX deficiency. A …

The Week in Review #21 Part 2 THE  MARKET (2) - Our remarks in the past Week in Review included our confidence that the attempts by negative investors to drive the market crazy will not deprive the market from its innate capability to sense and react to the authentic factors it usually reacts to . . . This content is for paid subscribers. Please click here to …

NEKTAR Nektar Therapeutics (NKTR) and Takeda Pharmaceutical (TKPYY) are collaborating to evaluate Nektar’s investigational medicine, NKTR-214, with Takeda’s investigational medicine, TAK-659, as a potential combination for multiple cancer settings.  NKTR-214 is an experimental immuno-stimulatory recognized to expand specific cancer-fighting T cells and natural killer (NK) cells directly into the cancer micro-environment. The drug also increases the expression of PD-1 on these immune cells. TAK-659 inhibits both spleen tyrosine kinase (SYK), which is involved in . . . …

The Week in Review #19 Targeting the Root-­Cause of Diseases - Investing in the Future is Currently the Best Way - Stopping aberrant genes from causing diseases has taken time to move from wishful thinking to reality. Through trial and error, various different approaches have been tested over the years, including attempts at genetic engineering... &nbsp . . . This content is for paid subscribers. …

Kite, a Gilead Company, and Sangamo Therapeutics Announce Collaboration to Develop Next-Generation Engineered Cell Therapies for Cancer Kite, a Gilead (GILD) company, and Sangamo Therapeutics (SGMO) announced a worldwide collaboration agreement for using Sangamo’s zinc finger nuclease (ZFN) technology platform for the development of next-generation ex vivo cell therapies in oncology. Kite will use Sangamo’s ZFN technology to modify genes to develop next-generation cell therapies for autologous and allogeneic use in treating …

The Week in Review #18 The Market - The stock market recuperated most of its losses while analysts preferred to abstain from forecasting and remain bystanders during the market volatility.  Although the causes of volatility have yet to be delineated, some philosophers in the field dared to attribute it as a market’s own mind’s judgment . . . This content is for paid subscribers. Please …

Prohost Letter #417 Private Lessons - These lessons are overdue and we had to do this homework before we go into further evaluations of our picks and introduction of new companies as we promised in the past issue. We believe that the Prohost readers, the fans and the critics should know how and why we pick our biotech stocks . . . This content is …

Prohost Letter #416 Part 2 2017 - A YEAR OF BREAKTHROUGH TECHNOLOGIES, BREAKTHROUGH PRODUCTS, IMPROVEMENTS ON PROMISING BREAKTHROUGH TREATMENTS, DECIPHERING IMPORTANT LIFE SCIENCE MYSTERIES, PINPOINTING BIOLOGICAL PATHWAYS FOR TREATMENT RESISTANCE, MIRACLE CURES, AND… A LOT OF DISTORTED NEGATIVE INTERPRETATIONS - Prioritizing our choices of breakthroughs has been a dilemma . . . This content is for paid subscribers. Please click here to subscribe or here to log …

Sangamo’s (SGMO) has abundant positive news since it offered proof of concept and feasibility of its zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation approach and other approaches. The most recent announcement was made early today by Sangamo and Pfizer (PFE) about a new collaboration. The cooperation aimed at developing a potential therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene. The firms will …

Gilead Sciences (GILD) has unsurprisingly agreed to acquire Cell Design Labs in a structured buy out valued for up to $567 million, including the shares of Cell Design Labs held by Kite, the CAR T firm that Gilead has recently acquired. Brian Atwood, President and CEO of Cell Design Labs, Inc., said, “Bringing our robust technology platforms under the Gilead umbrella, with its outstanding research …

The Week in Review #11 A New Era - Faster than the speed of light, the management of diseases is shifting from fast improvement to a total gigantic unprecedented change. Some of the conquering approaches towards providing long-­lasting cures look as if the outcome of magic rather than mere science . . . This content is for paid subscribers. Please click here to subscribe or here to log …

Important news has come from the FDA, which issued new guidelines to speed the introduction of treatments with human cells and tissues, including gene therapy. Cracking down on rogue clinics offering dangerous or unproven versions of gene therapy or stem cell therapy is also included in the report. Scott Gottlieb, the commissioner of the FDA stated that regenerative medicine and other important gene therapy treatments …

Yes, it is Sangamo’s (SGMO) zinc finger nuclease (ZFN) gene-editing technology, which for the first time in history enabled a patient to receive a treatment to edit the DNA of his cells directly inside the body. That was the news coming today from Sangamo. The Story Sangamo’s gene editing product known as SB-913 -- an investigational in vivo genome editing therapy was introduced for the first time …

The Week in Review #4 AMAZING STOCK REBOUNDING - We have already witnessed and wrote about the fierce rebounding of some of our selected stocks comprising: Exelixis (EXEL), Sangamo (SGMO), Array (ARRY), ImmunoGen (IMGN) and others . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

About Our New Section On Sunday, August 20, 2017 we will post our first “The Week in Review” article. This current week has carried bad news, political hypocrisy and a catastrophic terrorist attack on the people of Spain. The week started following war threatening words between the U.S. and North Korea; between the politicians around the world and in the U.S.; and between everybody and …

PTC THERAPEUTICS Why Buying Emflaza? Buying Emflaza, a steroid approved by the FDA for Duchenne muscular dystrophy (DMD) from Marathon Pharmaceuticals invited an intractable headache to PTC Pharmaceuticals’ shareholders and scrutiny by some law makers. The cause of the law makers’ involvement has been the Marathon’s pricing Emflaza at $89,000, but PTCT shareholders had several question marks revolving around the motive for buying this drug …

GENE EDITING Sangamo Biosciences: Is it a Comeback? Genome editing allows the creation of therapeutics that can modify the genome in a living cell, providing unprecedented reach that would help the creation of treatments for genetic diseases. The dream about emerging solutions through genome editing techniques, which could correct genetic mutations causing diseases seems to be turning into reality. Among the innovative firms in this …

Prohost Letter #389 Happy New Year 2016 Wishing You Good Health Peace of Mind Love And happiness  We expect 2016 to be the year that fulfills many of our great expectations about successfully managing intractable and life-threatening malignant and non-malignant disease. We expect the biotech firms to bring us more cures based on the maturation of genetic engineering processes following a lot of trial and …

Prohost Letter #388 TOPICS - 1. AN INDISPENSABLE SHORT INTRODUCTION 2. GENE EDITING 3. GENE THERAPY 4. CANCER IMMUNOTHERAPY 1. GENE EDITING Exon Skipping: Biomarin (BMRN); Sarepta (SRPT); These firms are waiting FDA approvals for Duchenne Muscular Dystrophy (DMD) Gene Editing Technology Competitors: Cellectis S.A. (CLLS); CRISPR; PTC Therapeutics (PTCT) Firms Using Different Approaches For DMD Treatment (see Below) Zink Finger Tech: Sangamo (SGMO); RNAi …