Fri.Oct 27, 2023

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STAT+: In major test for prime editing, scientists successfully correct mutations in monkeys

STAT

Prime Medicine said Friday it successfully used a new, ultra-versatile form of genetic surgery called prime editing to edit liver cells in monkeys. The results, presented at the European Society of Gene & Cell Therapy meeting in Brussels, are a major step for a technology that could transform treatment of numerous diseases. “I think the big celebration here is we’re showing, in primates, for the company, that we have a delivery system that is working and is safe,” said J

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SAEM Clinical Images Series: Intracranial Abnormality

ALiEM - Pharm Pearls

A 26-year-old male with no significant past medical history presented to the ED after slipping on wet pavement and hitting his head on the ground three hours prior. He endorsed a constant, achy 7/10 headache accompanied by nausea and photophobia. He denied vomiting, dizziness, diplopia, loss of consciousness, or seizures. Nothing made it better or worse.

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Emergency medicine is grappling with a surprising problem: The prospect of too many physicians

STAT

While much of the health care workforce is battling staff shortages, the emergency medicine field is trying to figure out how to manage a potential oversupply of physicians. The American College of Emergency Physicians predicted in a 2021 study that there would be a surplus of nearly 8,000 emergency physicians by 2030.

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The 2023 Red Jacket honorees

PharmaVoice

This year's inductees to the PharmaVoice 100 "hall of fame" are leaders who have been at the forefront — and will stay at the forefront — of industry change.

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Position Your Pharmacy for Expansion

Speaker: Chris Antypas and Josh Halladay

Access to limited distribution drugs and payer contracts are key to pharmacy expansion. But how do you prepare your operations to take the next step? Meaningful data: Collect and share clinical data regarding outcomes, utilization, and more Reporting: Limited distribution models require efficient tracking and reporting systems Workflows: Align workflows with specific pharma and payer contractual requirements For in-depth, expert insights on pharmacy expansion, watch this webinar from Inovalon.

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STAT+: FDA has some safety concerns about CRISPR-based sickle cell treatment, documents show

STAT

The Food and Drug Administration said on Friday that it has some safety concerns about an experimental CRISPR-based treatment for sickle cell disease, citing the methods used by its makers to evaluate the risk of inadvertently making unwanted changes to patients’ DNA. None of the concerns raised by the FDA on Friday, however, suggest the agency is reluctant to approve the treatment, called exa-cel, which is being developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

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Red Jacket: Ken Getz, a champion of clinical trials

PharmaVoice

Ken Getz has spent his career improving clinical research for the benefit of patients, the life sciences industry and other R&D stakeholders.

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More Trending

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Red Jacket: Dr. Jeremy Levin, a transformational leader

PharmaVoice

A consummate life sciences executive who has led with boldness and devotion, Dr. Jeremy Levin considers himself a participant as well as a leader.

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Opinion: Excited delirium: a deadly term finally laid to rest

STAT

George Floyd. Angelo Quinto. Elijah McClain. All three men were killed by lethal force inflicted by the police, and all three deaths were initially blamed on something called “excited delirium.” For years, law enforcement officers and other first responders were taught that people suffering from this so-called medical condition were uncontrollably strong, irrational, and required the most severe measures to be subdued.

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Red Jacket: Adele Gulfo, a commercialization guru

PharmaVoice

With a knack for melding science and patient-focused marketing techniques, the CEO of the biopharma commercial unit at Sumitomo Pharma America has notched blockbuster successes throughout her career.

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Opinion: The pink ribbon of breast cancer awareness needs a splash of blue for men

STAT

Everyone knows the universal symbol of breast cancer: the pink ribbon. Promoted by the Union for International Cancer Control , it pops up everywhere, from retail shops to government offices, each October for Breast Cancer Awareness Month. Police departments have pink ribbons on their vehicles to show solidarity; one town near me even has an entirely pink police cruiser.

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5 Reasons to Upgrade Your Pharmacy Management Software

Are you still using workarounds to manage your daily operations? To achieve peak performance, it's time to explore other options for specialty and infusion pharmacy software. Streamline pharmacy operations and improve clinical performance with automated processing, real-time data exchange, and electronic decision support. Download this helpful infographic to: Drive efficiency and patient adherence from referral receipt to delivery and ongoing care – all with our Pharmacy Cloud.

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Red Jacket: Helen Sabzevari, an immunotherapy pioneer

PharmaVoice

Precigen’s CEO is pushing for her next therapeutic breakthrough with innovative cell and gene therapy platforms.

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STAT+: Biden admin proposes fixes to No Surprises Act’s dispute resolution process

STAT

The Biden administration dropped a new proposal Friday designed to fix the controversial dispute resolution process that’s part of the federal surprise billing ban. Since the beginning of 2022, the No Surprises Act has shielded patients in most cases against bills from out-of-network providers for care delivered at in-network facilities. But the behind-the-scenes battles between those providers and health insurers over what that care should cost have gotten ugly.

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Red Jacket: Dr. Mark Goldberg, a clinical innovator

PharmaVoice

Dr. Mark Goldberg has continually been at the cutting edge of technologies and ideas, including now as chairman and CEO of Allucent.

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STAT+: ARPA-H head Renee Wegrzyn on the agency’s new Cambridge hub and plans for cancer research

STAT

CAMBRIDGE, MASS. — In an area long known as an epicenter of innovation and investment in health care, the Advanced Research Projects Agency for Health (ARPA-H) officially launched its investor hub yesterday that will be dedicated to commercializing its research on biomedical solutions. ARPA-H, which boasts a $1.5 billion budget and is modeled after the Pentagon’s science office DARPA, is rolling out a “hub-and-spoke” structure, with two other hubs, in the D.C. suburbs

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CAR-TCR 2023 – Matt Lakelin

pharmaphorum

At CAR-TCR in Boston last month, pharmaphorum’s Jonah Comstock caught up with Matt Lakelin, co-founder of TrakCel, a software company that helps makers of advanced therapies manage their supply chain.

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Regeneron gene therapy improves hearing in child

STAT

Want to stay on top of the science and politics driving biotech today?  Sign up  to get our biotech newsletter in your inbox. Hi! Today we see that prime editing works nicely in monkeys, learn more about the potential new bill to speed treatments for life-threatening diseases, and find that a Regeneron (formerly Decibel) therapy may restore hearing in children.

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Bristol Myers’ neuroscience research head explains the big pharma’s return to brain drugs

BioPharma Dive

In a wide-ranging interview, Bristol Myers’ Ken Rhodes talked biomarkers, diseases of interest and why the company is far from done with neuroscience-focused deals.

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STAT+: Pharmalittle: Sanofi plans to spin off its consumer health biz; EMA says no evidence GLP-1 drugs linked to thyroid cancer

STAT

And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is rather modest. We plan to promenade with the official mascots, tidy up the grounds, and maybe have a listening party with Mrs. Pharmalot. The rotation may include this , this , this and this.

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Parkinson’s UK awards over £1.8m for nine research projects

Pharma Times

Around 145,000 people in the UK are living with Parkinson’s disease - News - PharmaTimes

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STAT+: Up and Down the Ladder: The latest comings and goings

STAT

Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others.  That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going. And here is our regular feature in which we highlight a different person each week.

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Sanofi to divest consumer unit, joining pharma industry shift

BioPharma Dive

The restructuring came as the French company forecast an earnings drop in 2024, prompting a stock selloff that erased about $20 billion in market value.

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What You Need to Know About the New COVID-19 Vaccines

Community Pharmacy

While COVID-19 might not be as pressing of a problem as it was during the peak of the pandemic, the virus and its many variants still pose significant health risks that require updated vaccine protection to combat. As they have in years past, another surge of cases has been anticipated by experts as we enter the holiday season. Along with well-known variants like Omicron, 2023 has seen other COVID mutations like XBB.1.5. rise, which previous year’s vaccines were not designed to protect against.

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Levels of obesity twice as high among children in most deprived areas

The Pharmacist

Levels of obesity in reception-aged children living in the most deprived areas of England are more than double those in the least deprived areas, new data has shown. According to NHS Digital, levels of obesity among children aged four-to-five in the worst-off areas of England stood at 12.4%, compared to 5.8% in more well off areas. […] The post Levels of obesity twice as high among children in most deprived areas appeared first on The Pharmacist.

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Regulators develop five principles for machine learning-enabled medical devices  

Pharma Times

The new principles will remove the regulatory burden for MLMD manufacturers - News - PharmaTimes

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ThermoGenesis opens cell and gene therapy facility in Sacramento, US

Pharmaceutical Business Review

Designed to serve as an incubator facility, the new modern facility includes ISO 7 cGMP cleanroom suites, as well as research and development (R&D) labs. The ReadyStart cGMP Cleanrooms and IncuStart Wet Labs offer a cleanroom environment, equipment, and services for R&D and cGMP manufacturing of cell and gene therapies and other life science products.

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Sustainable biosimilar competition in Europe: can it be achieved?

European Pharmaceutical Review

According to The Biosimilar Medicines Group, part of Medicines for Europe, a new report by IQVIA indicates there is a window of opportunity for the EU to foster future-proof biosimilar competition. This publication has come in wake of the EU actively reviewing its Pharmaceutical Legislation. Julie Marechal-Jamil, Director Biosimilar Policy & Science at Medicines for Europe stated the report identified “many policy shortcomings that must be addressed now to deliver access to medicines in the

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SETU secures funding from HNIs

Express Pharma

SETU aims to grow their business 30 times over the next five years, capitalising on strong category and channel growth Setu Nutrition (SETU), direct-to-consumer supplements founded by Nihaal Mariwala and backed by the Mariwala Family of OmniActive Health Technologies, has secured substantial funding through investments made by High Net Worth Individuals (HNIs) and family offices, including tennis icon Sania Mirza.

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NHS launches blood pressure service finder

The Pharmacist

An online tool to help patients find a pharmacy offering the NHS Blood Pressure Check Service has been launched for patients in England. As part of the move, pharmacy contractors have been reminded to ensure their pharmacy profile on the NHS profile manager is up to date, including details of whether they provide the service. The new […] The post NHS launches blood pressure service finder appeared first on The Pharmacist.

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New Bill could enhance UK clinical trial regulation

European Pharmaceutical Review

A report published by the Policy Exchange has highlighted how The Future Clinical Trials Bill could improve the UK’s approach to the establishment and regulation of clinical trials. The new Bill is part of a range of proposed policies for implementation under the new legislative programme that will be set out in the final session of this Parliament.

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Monoclonal antibodies for Nipah treatment to be developed indigenously: Kerala Health Minister

Express Pharma

The minister said the monoclonal antibodies were presently being imported from Australia where they are made according to the Nipah variant present there The monoclonal antibodies which are the only treatment against Nipah infection will be developed indigenously to combat the deadly virus, Kerala Health Minister Veena George said on Wednesday. George revealed that a decision had been taken by two national and a Kerala government institute to develop monoclonal antibodies.

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UK NICE recommends use of AbbVie’s Parkinson’s treatment 

Pharmaceutical Technology

The UK NICE has recommended the use of AbbVie’s foslevodopa–foscarbidopa (Produodopa) for the treatment of advanced Parkinson’s disease.

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Independent prescribing training for existing workforce ‘biggest challenge’ for pharmacists

The Pharmacist

Independent prescribing (IP) training for the existing workforce was considered the biggest challenge facing the profession in our exclusive survey of community and practice pharmacists. Respondents were asked what they thought were the biggest challenges facing pharmacists at the moment, with the ability to select multiple options from a list or add another option.

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GoodRx Announces New Way for Consumers To Access Sanofi’s Lantus for $35 at Over 70,000 Pharmacies Nationwide

Drug Channels

Today’s guest post is from Dorothy Gemmell, Chief Commercial Officer at GoodRx. Dorothy explains the innovative new collaboration between GoodRx and Sanofi. Under the program, the out-of-pocket cost for Sanofi's Lantus will be capped at $35 at any pharmacy that accepts GoodRx. This program will increase affordabiliy and broaden access for people living with diabetes.

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FDA staff focus on ‘off-target’ risk in review of Vertex, CRISPR sickle cell therapy

BioPharma Dive

Documents released ahead of a Tuesday advisory meeting show agency scientists to be generally convinced by the treatment’s benefit, a sign analysts read as positive.

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Roche enters into a definitive agreement to acquire Telavant including rights to novel TL1A directed antibody (RVT-3101) for the treatment of inflammatory bowel disease from Roivant

World Pharma News

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced the entry into a definitive agreement to acquire Telavant Holdings, Inc. (Telavant), a Roivant company, owned by Roivant Sciences Ltd. and Pfizer Inc. The agreement includes the development, manufacturing and commercialisation rights in the US and Japan for Telavant's RVT-3101, a novel TL1A directed antibody.

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