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Alnylam Initiates Phase I Clinical Study for ALN-TTRsc, a Subcutaneously Administered RNAi Therapeut

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By Business Wirevia The Motley Fool

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Alnylam Initiates Phase I Clinical Study for ALN-TTRsc, a Subcutaneously Administered RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR)

– ALN-TTRsc is Company’s First Subcutaneously Delivered RNAi Therapeutic to Enter Clinical Trials –

– Data from Trial Expected Mid-2013 –

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Alnylam Pharmaceuticals, Inc. (NAS: ALNY) , a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). ATTR is caused by mutations in the TTR gene which cause abnormal amyloid protein deposits to accumulate in various tissues including peripheral nerves and heart, resulting in neuropathy and/or cardiomyopathy. ATTR represents a major unmet medical need with significant morbidity and mortality; familial amyloidotic polyneuropathy (FAP) affects approximately 10,000 people worldwide and familial amyloidotic cardiomyopathy (FAC) affects at least 40,000 people worldwide. ALN-TTRsc, which is being developed for the treatment of FAC, is a subcutaneously administered RNAi therapeutic that comprises an siRNA conjugated to a GalNAc ligand that enables receptor-mediated delivery to the liver. ALN-TTRsc is the first GalNAc-siRNA and the first subcutaneously delivered, systemic RNAi therapeutic to enter clinical development stages.

“RNAi therapeutics hold great promise for the treatment of ATTR since they have demonstrated rapid, potent, and durable knockdown of TTR, the disease-causing protein. We are advancing what we believe to be the industry leading effort in ATTR; this includes ALN-TTRsc for the treatment of FAC and ALN-TTR02 for the treatment of FAP which is currently enrolling patients in a Phase II trial,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “The start of this Phase I trial with ALN-TTRsc represents the first for an RNAi therapeutic that utilizes our proprietary GalNAc conjugate delivery platform. It also marks the first subcutaneously delivered, systemic RNAi therapeutic for the industry. We look forward to the continued advancement of ALN-TTRsc for the treatment of FAC.”

The Phase I trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects. The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc. Secondary objectives include assessment of clinical …read more
Source: FULL ARTICLE at DailyFinance

Automobiles

Quick Play: <i>Table Top Racing</i>

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By Seyth Miersma

Tabletop Racing splash screen

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Studio Playrise Digital has recently launched a clever new micro-scaled racing game for Apple’s App Store, called Table Top Racing ($2.99). As the name indicates, the basic theme for the iPhone and iPad app is that of a kid staging imaginative “races” with toy cars – all along the surfaces of handy, obstacle-dotted table tops. After spending a few days with Table Top Racing, here’s what we thought.

Play Notes

  • The overall look and feel of the game is chunky, charming and easy to understand. Menus are simple and streamlined, and designed so that you’re pretty much always just one step away from your garage, where all of the important car selecting and tuning happens. The graphics are bright, clear and clean, though hardly cutting edge in the ever-more sophisticated world of tablet/smart phone gaming.
  • Racing action is very simple to catch on to. Races happen in a few different formats, but basically this is a Mario Kart-lite experience. Drive wacky cars, fire weapons or set traps along the track, try to survive the race and finish in first place.
  • Steering is accomplished by way of right/left buttons on the extreme sides of the screen. We played Table Top Racing exclusively on the iPad for this test, and found the steering buttons to be a bit on the small side. It’s too easy to “miss” the button zone and then miss your turn as a result. Don’t expect your Micro Machine-style racer to handle with realistic physics, either. This wasn’t our expectation, and the driving is perfectly satisfying for a game of this type with such stylized handling.
  • Difficulty feels spot-on in this app. It’s easy enough to get started and win a few races off the bat, and earning enough money to keep your racer competitive isn’t too challenging in the early going. By the later Championships, the going is reasonably tougher. Of course, TTR allows for you to make purchases of in-game coins using real money, too, so you can buy your way into the top-spec cars whenever you get too frustrated.
  • With all of that said, we would def finitely consider this to be an “all-ages” app. Kids should have no problem with the straightforward controls; adults will find even the enough variation in difficulty to make this enjoyable.
  • At $2.99, this feels like a good buy from the App Store. If our weekend’s worth of testing is any indication, you’ll spend 10 hours or more just working through the Championships, and a few more than that completing every race and buying every vehicle. This is a really good choice if you’ve got a few long flights, train rides, etc. staring you down, with nothing to while away the time.

In addition to our full-length reviews of big-name console racing games, we thought it would be useful to publish brief Quick Play reviews of interesting new racing titles. If you’ve got a tip about a new racing, driving or otherwise automotive-themed …read more
Source: FULL ARTICLE at Autoblog