Huntington drug successfully lowers levels of disease-causing protein

medicalxpress | May 06, 2019

An international clinical trial has found that a new drug for Huntington disease is safe, and that treatment with the drug successfully lowers levels of the abnormal protein that causes the debilitating disease in patients. In a study published today in the New England Journal of Medicine, researchers from UBC and their colleagues have demonstrated for the first time that the drug, IONIS-HTTRX (now known as RO7234292) successfully lowered levels of the mutant huntingtin protein—the toxic protein that causes Huntington disease—in the central nervous system of patients. "This is a tremendously exciting and promising result for patients and families affected by this devastating genetic brain disorder," said Dr. Blair Leavitt, neurologist and director of research at the Centre for Huntington Disease at UBC. "For the first time, we have evidence that a treatment can not only decrease levels of the toxic disease-causing protein in patients, but that it is also safe and very well tolerated."

Spotlight

Merck & Co, known as MSD outside of the United States and Canada, is a global pharmaceutical company whose mission is to discover, develop and provide innovative products and services that save and improve lives around the world.

Spotlight

Merck & Co, known as MSD outside of the United States and Canada, is a global pharmaceutical company whose mission is to discover, develop and provide innovative products and services that save and improve lives around the world.

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BUSINESS INSIGHTS, PHARMA TECH

Specialty Pharma Expert Adopts Name of Global Parent to Reflect Breadth of Offer That Brings a Rare and Special Perspective to the Market

Businesswire | April 26, 2023

Global healthcare and business consulting services provider, Terebellum, has chosen to adopt the name of its global parent, AscellaHealth, to reflect the scale of its operations and breadth of expertise. The business specialises in providing end-to-end solutions to pharmaceutical manufacturers and other industry stakeholders to streamline product commercialisation, improve medication access, reduce costs and enhance patient outcomes for specialty and rare disease patients. Based in Dublin, Ireland and Manchester, UK, former Terebellum provides pre-commercialisation and market access support, supply chain logistics, distribution and fulfilment and unique pharmaceutical financial services to UK and European pharmaceutical manufacturers and healthcare organisations. Adopting the brand name of its parent company, AscellaHealth, will enable the specialty pharma expert to demonstrate its full range of expertise, international insight and distinctiveness of offer. Upon Terebellum’s transition to the AscellaHealth brand identity in its market-facing activities, the company will continue to maintain current business operations, client relationships and dedication to patient-centric care. Craig Caceci, Managing Director at AscellaHealth EU/UK, said: “It’s our strong belief that to optimise outcomes in specialty healthcare a specialised approach is needed, due to the specific challenges associated with rare conditions. “The AscellaHealth name provides an opportunity to highlight how we are perfectly positioned to offer uniquely tailored solutions across the globe to ensure streamlined medication access and the best possible treatment journey for rare and orphan disease patients.” In a recent customer survey carried out by AscellaHealth, 100 percent of respondents agreed that the organisation presents unique value in the market and is uniquely qualified to address their needs. Craig adds: “Supporting patients, life science manufacturers, payers and providers, we bring expertise and care to every requirement to develop and implement novel approaches that ensure patients with rare diseases can get the treatment they need, when they need it most. “Our new brand tagline, ‘a rare and special perspective’, captures our dedication to applying knowledge and expertise to improve health outcomes of rare disease and specialty patients, perfectly.” AscellaHealth, co-founded by Dea Belazi, President & CEO and Bill Oldham, Chairman & CFO in 2013, employs nearly 200 employees worldwide to provide integrated end-to-end solutions that span the entire pharmaceutical lifecycle and beyond, supporting all segments of the specialty pharma industry. Former Terebellum joins US-based pharmacy distribution and patient-management organisation, Optime Care, in adopting the name of parent AscellaHealth, to form one globally-recognised brand for specialty pharma expertise. About AscellaHealth LLC AscellaHealth, a global Healthcare & Specialty Pharmacy solutions organisation, serving patients, life sciences manufacturers, payers, and providers, offers a comprehensive portfolio of uniquely tailored, tech-enabled services supporting complex, chronic conditions or rare diseases that require specialty medications and/or cell and gene therapies. A recipient of numerous industry awards for innovation, and a NASP Strategic Channel Partner of the Year award winner, AscellaHealth’s best-in-class, patient-centric approach is built upon proprietary technology processes for novel programs and services to support the launch of specialty medications and proactively address multiple challenges, optimise clinical health outcomes, and improve quality of life for this patient population. AscellaHealth brings a rare and special perspective to all stakeholders.

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PHARMA TECH

XtalPi Announces Collaboration with Lilly, Using AI + Robotics to Uncover First-in-class Therapeutics

PRNewswire | May 31, 2023

Today, XtalPi Inc., a pioneering pharmaceutical technology company powered by artificial intelligence (AI) and automation, announced an AI drug discovery collaboration of up to $250 million in upfront and milestone payments with Eli Lilly and Company. The collaboration will leverage XtalPi's integrated AI capabilities and robotics platform to de novo design and deliver drug candidates for an undisclosed target. XtalPi has established itself as an industry leader in combining mutually informative AI "dry lab" algorithms with large-scale "wet lab" robotics to empower pharmaceutical innovation. Building upon existing success, XtalPi will work closely with the Lilly team to harness the power of its proven one-stop AI drug discovery solution to deliver a novel compound, which Lilly will pursue in clinical and commercial development. XtalPi's ID4Inno™ small-molecule drug discovery platform touts a tightly interwoven trifecta of AI, autonomous labs, and expert domain knowledge that can generate and investigate a target-specific mega chemical space and quickly zero in on the most promising lead series. Each group of synthesized molecules is tested using XtalPi's internal biochemical, cellular, pharmacodynamic, and pharmacokinetic assay capabilities, feeding project-specific R&D data into generative AI models in iterative design-make-test-analyze cycles until a candidate with optimal drug properties emerges. XtalPi's fleet of hundreds of autonomous robotic workstations can carry out precise and energy-efficient parallel chemical synthesis and assays 24/7. This high-quality real-world data generated with speed and at scale, combined with over 500 AI models and quantum physics algorithms, continuously enhance XtalPi's capability to predict and authenticate novel drug candidates with greatly accelerated timeframes and reduced experimental costs. XtalPi's AI + robotics platform is supercharging the shift of pharmaceutical R&D from labor-intensive trial-and-error research to a computation and automation-intensive model, empowering scientists to achieve more with less. Serving over 200 pharmaceutical clients and research institutions from around the world, XtalPi is now bringing its proprietary autonomous lab to Cambridge, MA as its drug discovery business continues to grow. Dr. Jian Ma, CEO of XtalPi, says, "With a closed loop of AI and quantum physics algorithms working in sync with the data factory of large-scale robotics experiments, XtalPi is uniquely equipped to tackle challenging novel targets. We are honored that Lilly has chosen XtalPi's AI + robotics drug R&D platform as a partner in achieving more fruitful pharmaceutical innovation and bringing much-needed treatments to patients worldwide." About XtalPi XtalPi is a pharmaceutical technology company powered by AI and robotics to improve the speed, scale, novelty, and success rate of drug discovery and development. With operations in both China and the U.S., XtalPi has pioneered a new model for drug R&D that leverages a tightly interwoven trifecta of AI, autonomous labs, and expert domain knowledge. Its integrated AI "dry lab" and large-scale robotics "wet lab" capabilities form an iterative feedback loop that empowers continuous innovation to address the unmet needs of patients worldwide.

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PHARMACY MARKET, PHARMA TECH

PANTHERx® Rare Launches Drug for APDS

prnewswire | March 31, 2023

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