STAT
DECEMBER 8, 2023
The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine , called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics.
PharmaVoice
DECEMBER 8, 2023
A look at the fast-evolving pipeline for gene editing therapies.
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STAT
DECEMBER 8, 2023
For 39 years, I have lived with sickle cell disease. As of late, the sickle cell community has heard a lot of excitement about gene therapy’s potential to be the cure we’ve all been waiting for. That potential took a step toward fulfillment Friday when the Food and Drug Administration approved Casgevy from Vertex Pharmaceuticals’ and Lyfgenia from Bluebird Bio, both gene therapy treatments for people with sickle cell.
ALiEM - Pharm Pearls
DECEMBER 8, 2023
A 45-year-old male status-post right nephrectomy secondary to a renal mass presented to the emergency department with right-sided flank pain. He endorsed low-grade intermittent right-sided flank pain since the nephrectomy one year prior, associated with an increasingly enlarging mass extending laterally from his right abdomen. Over the course of the past several days, the mass had become larger and more painful.
Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.
The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven
STAT
DECEMBER 8, 2023
Editas Medicine, CRISPR Therapeutics, and Intellia Therapeutics were all started in the 2013-2014 time frame with the same goal of turning the nascent CRISPR gene-editing technology into medicines. With the approval of Casgevy , the world’s first gene-edited therapy and a potential cure for sickle cell disease, CRISPR Therapeutics won the race.
BioPharma Dive
DECEMBER 8, 2023
In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.
Pharmacist Digest brings together the best content for pharmacists from the widest variety of industry thought leaders.
Pharma Times
DECEMBER 8, 2023
One-third of patients who received Pfizer’s COVID-19 vaccine had unintended immune responses - News - PharmaTimes
STAT
DECEMBER 8, 2023
Research universities are voicing concerns over some proposed changes to the process for reviewing scientific misconduct allegations, citing worries that new government requirements would hobble their own independent procedures and constitute a huge administrative burden. Nearly two decades since it last released research misconduct rules, the U.S. Office of Research Integrity (ORI) is ready to update its requirements for ensuring the scientific standards of projects funded by the U.S.
Safe Biologics
DECEMBER 8, 2023
On November 30, ASBM and the Generics and Biosimilars Initiative (GaBI) hosted I nterchangeable Designation for Biosimilars- Ensuring Continuity of Patient Care: Upholding Interchangeability Status for Biosimilars. The webinar was the fourth in a series covering key health policy issues. The most recen t of the webinars, hosted July 29th, examined the negative impact of the Inflation Reducation Act’s Medicare drug price setting provisions.
STAT
DECEMBER 8, 2023
In a win for Vertex Pharmaceuticals, a Colorado state board voted not to place the company’s best-selling cystic fibrosis medication on a list of drugs for which payment limits will be set for most residents, including those whose insurance is covered by a government agency or a commercial health plan. The Colorado Prescription Drug Affordability Board voted unanimously that the expensive medicine, which carries a wholesale price of about $310,000 in the U.S. before any rebates or discoun
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Pharma Times
DECEMBER 8, 2023
The project will explore the biology of human autoreactive plasma cells - News - PharmaTimes
STAT
DECEMBER 8, 2023
“Can I tell you what I really think about what’s going on?” she asks, looking down and clutching her coffee, her right sneaker tapping. I had been fairly sure about two things ahead of our session: one, that she would talk about the Conflict, and two, that I would dislike what she was going to say. I gulp what I hope is a hidden gulp, every inch of me wanting to tell her no actually, can we please not go there and just stick with your relationship unhappiness?
BioPharma Dive
DECEMBER 8, 2023
Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1 million.
STAT
DECEMBER 8, 2023
Health officials on Friday released the first nationally representative estimate of how many U.S. adults have chronic fatigue syndrome: 3.3 million. The Centers for Disease Control and Prevention’s number is larger than previous studies have suggested, and is likely boosted by some of the patients with long COVID. The condition clearly “is not a rare illness,” said the CDC’s Dr.
Speaker: Joe Sharpe and James Carlson
Payroll optimization can be one of the most time-consuming and complex factors of small business management. Yet, organizations that crack the code on streamlining employee compensation often discover innovative avenues for growth. With the right strategies in place, outsourcing and streamlining payroll processes can result in substantial time and resource savings.
pharmaphorum
DECEMBER 8, 2023
Pfizer’s initiative to return clinical trial data to patients Mike.
STAT
DECEMBER 8, 2023
Research universities are voicing concerns over some proposed changes to the process for reviewing scientific misconduct allegations, citing worries that new government requirements would hobble their own independent procedures and constitute a huge administrative burden. Nearly two decades since it last released research misconduct rules, the U.S. Office of Research Integrity (ORI) is ready to update its requirements for ensuring the scientific standards of projects funded by the U.S.
pharmaphorum
DECEMBER 8, 2023
PharmStars digital health accelerator names fall 2023 grads Phil.
STAT
DECEMBER 8, 2023
On Thursday, the Medicare Payment Advisory Commission reviewed Medicare performance data and concluded that despite doctors’ protests that their pay hasn’t kept up with inflation over the last 20 years, doctors shouldn’t get more than a minor raise for 2025. On the other hand, the committee admitted hospitals could use a larger increase in payment rates.
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World Pharma News
DECEMBER 8, 2023
Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
STAT
DECEMBER 8, 2023
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.
BioPharma Dive
DECEMBER 8, 2023
The setbacks for Merck demonstrate the challenge drug developers face in improving on Keytruda, which has brought in at least $18 billion so far this year for its maker.
STAT
DECEMBER 8, 2023
Understand how science, health policy, and medicine shape the world everyday. Sign up for our Morning Rounds newsletter here. Institutions push back on scientific misconduct rules Investigating scientific research misconduct is necessarily a heavy lift for universities and other institutions. Many are now saying that proposed changes from the U.S.
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.
pharmaphorum
DECEMBER 8, 2023
Africa-led HIV vaccine trial ends in failure Phil.
STAT
DECEMBER 8, 2023
Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. Today, we talk about the much-awaited CRISPR-based drug Casgevy, discuss the new Gavi initiative that plans to dramatically ramp up vaccine manufacturing in Africa, and more. Have a nice weekend!
PharmExec
DECEMBER 8, 2023
FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals' and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease.
pharmaphorum
DECEMBER 8, 2023
Why pharma needs to leave digital transformation behind and embrace digital business Mike.
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European Pharmaceutical Review
DECEMBER 8, 2023
AbbVie has signed a definitive agreement to acquire Cerevel Therapeutics and its neuroscience pipeline – including antipsychotic emraclidine – for approximately $8.7 billion. Cerevel’s pipeline includes multiple clinical-stage and preclinical candidates with potential across several diseases including schizophrenia, Parkinson’s disease, and mood disorders.
pharmaphorum
DECEMBER 8, 2023
Life sciences industry predictions 2024: Curavit Mike.
Pharmaceutical Commerce
DECEMBER 8, 2023
Study analyzes and predicts how conditions in research and development can be predicted via computer simulation.
The Pharmacist
DECEMBER 8, 2023
NHS England is not confident IT solutions will be in place for the planned 31 January rollout of Pharmacy First, senior officials have revealed. The Government had already said the rollout date was subject to the ‘appropriate digital systems being in place’ to support the services. However, during a public meeting yesterday NHS England’s board was told […] The post NHS England board questions if IT will be in place for January Pharmacy First rollout appeared first on The Pharmacist.
pharmaphorum
DECEMBER 8, 2023
Spyre raises $180m to advance IBD antibody pipeline Phil.
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