Michael Tattory

Silencing Novel Target Genes: A New Strategy for Lipid Lowering

Two more novel lipid-lowering therapies based on silencing specific genes involved in lipid metabolism have shown promising preliminary results in initial clinical trials. The idea of using small pieces of RNA, known as small interfering RNA (siRNA) specific for a certain gene to switch off the activity of that gene is a new area for

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Plano-based drug developer Reata raises $505 million, expects to double to 400 employees by end of 2020

Plano-based drug developer Reata Pharmaceuticals Inc. raised more than $505 million in a stock sale Monday to speed its transition from a clinical-stage biotech startup to a company that makes treatments for life-threatening diseases. The publicly-traded company will use the money to register and commercialize its first two treatments: one for Alport syndrome, a genetic

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Novel RNA interference agents improve lipid profiles in healthy volunteers

Two novel RNA interference agents, one targeting apolipoprotein C-III and one targeting angiopoietin-like protein 3, improved various lipid parameters in early studies with healthy volunteers, according to new data presented at the American Heart Association Scientific Sessions. One agent targeting apolipoprotein C-III (ARO-APOC3, Arrowhead) was associated with reductions in serum APOC3 and improvements in other

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FDA Approves Talicia To Treat Superbug Linked To Stomach Cancer

The FDA’s decision to approve Talicia could have far-reaching impact, as this bug infects millions of American adults a year and is one of the strongest known causes of stomach cancer. Talicia is a three-drug combination of omeprazole, a proton pump inhibitor, amoxicillin, a penicillin-class antibacterial, and rifabutin, a rifamycin antibacterial. It is indicated for

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Tissue Donor Tuly Gonzalez’s Legacy Continues Through The 8 Lives She Impacted

Family and friends gathered at the University of Miami Life Science and Technology Park Wednesday morning to remember Tuly Gonzalez on what would have been her 32nd birthday. She died 11 years ago but the gathering was not just to remember her, but to also celebrate the lives she has impacted though her donation.

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Novel therapy safe, effective in acute, severe alcoholic hepatitis

In this exclusive video from The Liver Meeting 2019, Tarek Hassanein, MD, FAASLD, professor of medicine at UC San Diego Health and medical director of Southern California GI and Liver Centers, discusses results of a phase 2a study, which demonstrated that DUR-928 was well-tolerated and effective in patients with acute alcoholic hepatitis.

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AASLD: Durect’s alcoholic hepatitis med repairs liver, cuts mortality in phase 2

Hepatitis used to be the talk of the town at The Liver Meeting, the annual gathering of the American Association for the Study of Liver Diseases. These days, the conversation is turning to nonalcoholic steatohepatitis (NASH), but its cousin alcoholic hepatitis (AH) isn’t getting much attention. Durect is trying to change that.

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First Results Out for Off-the-Shelf CAR-T Therapy in Solid Tumors

The interim results come from an ongoing phase I trial in patients with colorectal cancer. Of the 12 patients given Celyad’s CAR T-cell immunotherapy so far, six have seen their tumors shrink. The therapy has also shown no evidence of causing graft-versus-host disease, a condition where foreign immune cells attack the host body. Celyad’s CAR T-cell immunotherapy is

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Why It Could Soon Be Time To Take Profits In This Highflying Biotech Stock

Arrowhead Pharmaceuticals (ARWR) and Johnson & Johnson (JNJ) said Friday they eradicated nearly all traces of hepatitis B virus in a Phase 2 study — sending Arrowhead stock careening toward a record high. On the stock market today, Arrowhead stock jumped 6.3% to 42.10. On Monday, shares hit a record high in intraday action of 42.29. Arrowhead stock broke out on Oct.

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Vision Tech: Several Gene Therapies for Blindness Reach Clinical Trials

ProQR Therapeutics is a Netherlands-based company currently researching RNA therapies to inhibit or counteract the harmful effects of the defective genes associated with the above retinal diseases. How does that work? As you may recall from biology class, genes are the building blocks for the proteins that build and maintain our bodies.

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Cell and Gene Therapies Necessitate New Lot Release Test Methods

Product-release testing is required for all drug products to ensure their quality and safety. For most conventional small-molecule and biologic drugs, batches are held until the release testing results are received and confirm the product is within specifications. Cell and gene therapies and some of their components (e.g., viral vectors), however, often must be used

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This Biotech Stock With A 221% Gain In 2019 Is Ready For ‘Prime Time’

Arrowhead Pharmaceuticals (ARWR) is using a technology called RNAi to tackle two colossal hurdles in treating genetic diseases. And it’s helping Arrowhead stock to finally realize profits after the biotech company operated for years in the red. RNAi, or RNA interference, is a method of silencing genes. Some genetic diseases cause the overproduction of troublesome

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What Makes A Biopharma Serial Entrepreneur Tick? Part 1

Biopharmaceutical executives, especially those who have founded their own companies, never cease to amaze me. Why? Well for starters, the process of starting with nothing but an idea and then pulling together all the necessary resources to build a company capable of developing and delivering a product to patients is an amazing logistical challenge. It

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Broadening the Applicability of CAR-T Immunotherapy to Treat the Untreatable

In the early 2000s, chimeric antigen receptor T (CAR-T) cells emerged on the scene with promise as a revolutionary cancer treatment. However, several key challenges and, in particular, downsides related to manufacturing, continue to hamper broad adoption of this therapeutic approach. In addition, existing CAR-T cell therapies are based on autologous approaches (i.e. using the own

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ProQR announces rare pediatric disease designation, phase 1/2 results for sepofarsen

Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics. Sepofarsen, an RNA-based oligonucleotide, is designed to address the underlying cause of LCA10 in the CEP290 gene. With the designation, the drug will receive priority review by the FDA, and it could

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Biotech Says It Hurdled A Genetic Barrier, But Shares Gave Up A Breakout

Arrowhead Pharmaceuticals stock flirted with a breakout Friday after the biotech company said it’s experimenting with drugs that will “silence” multiple troublesome genes simultaneously. Previously, that was impossible, Chief Executive Christopher Anzalone said late Thursday on CNBC’s Mad Money. Arrowhead Pharmaceuticals (ARWR) is among a slew of biotech companies using technology called RNA interference to silence genes responsible for creating

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Eye Drops To Reverse Vision Loss One Step Closer To Market

Eye drops that can reverse poor vision? It may sound like science fiction, but one Israeli company is aiming to bring this product to market. The drops passed their Phase 2b clinical trial earlier this month, meaning they’ve proven to improve farsightedness, the inability to see or read nearby objects, and are highly tolerable. Although results

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