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Modified CRISPR-based enzymes improve the prospect of inserting entire genes into the genome to overcome diverse disease-causing mutations

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Research
17 January 2023
Many genetic diseases are caused by diverse mutations spread across an entire gene, and designing genome editing approaches for each patient’s mutation would be impractical and costly.

Investigators at Massachusetts General Hospital (MGH) recently developed an optimized method that improves the accuracy of inserting large DNA segments into a genome.

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Study identifies potential new approach for treating lupus

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Research
13 January 2023
Targeting iron metabolism in immune system cells may offer a new approach for treating systemic lupus erythematosus (SLE) - the most common form of the chronic autoimmune disease lupus.

A multidisciplinary team of investigators at Vanderbilt University Medical Center has discovered that blocking an iron uptake receptor reduces disease pathology and promotes the activity of anti-inflammatory regulatory T cells in a mouse model of SLE.

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Nanotechnology may improve gene therapy for blindness

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Research
11 January 2023
Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness.

A collaborative team of researchers with Oregon Health & Science University and Oregon State University have developed an approach that uses lipid nanoparticles - tiny, lab-made balls of fat - to deliver strands of messenger ribonucleic acid, or mRNA, inside the eye.

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Incurable liver disease may prove curable

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Research
05 January 2023
Research led by Associate Professor Duc Dong, Ph.D., has shown for the first time that the effects of Alagille syndrome, an incurable genetic disorder that affects the liver, could be reversed with a single drug. The study, published in the Proceedings of the National Academy of Sciences, has the potential to transform treatment for this rare disease and may also have implications for more common diseases.
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Scientists develop a cancer vaccine to simultaneously kill and prevent brain cancer

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Research
04 January 2023
Scientists are harnessing a new way to turn cancer cells into potent, anti-cancer agents. In the latest work from the lab of Khalid Shah, MS, PhD, at Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, investigators have developed a new cell therapy approach to eliminate established tumors and induce long-term immunity, training the immune system so that it can prevent cancer from recurring.
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Time-restricted eating reshapes gene expression throughout the body

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Research
03 January 2023
Numerous studies have shown health benefits of time-restricted eating including increase in life span in laboratory studies, making practices like intermittent fasting a hot topic in the wellness industry. However, exactly how it affects the body on the molecular level, and how those changes interact across multiple organ systems, has not been well understood.
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COVID-19 vaccines, prior infection reduce transmission of Omicron

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Research
02 January 2023
Vaccination and boosting, especially when recent, helped to limit the spread of COVID-19 in California prisons during the first Omicron wave, according to an analysis by researchers at UC San Francisco that examined transmission between people living in the same cell.
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More Pharma News ...

  1. Enzyme that protects against viruses could fuel cancer evolution
  2. High levels of ammonia in colon tumors inhibits T cell growth and response to immunotherapy
  3. COVID-19 booster increases durability of antibody response, research shows
  4. Artificial DNA kills cancer
  5. Large, real-world study finds COVID-19 vaccination more effective than natural immunity
  6. Killing pancreatic cancer with T cells that supercharge themselves
  7. Drug discovery offers potential treatment for common kidney disease
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Business & Industry

  • Insilico Medicine brings AI-powered "ChatPandaGPT" to its target discovery platform
  • BlueRock Therapeutics to incorporate wearable and invisible contactless digital health technologies from Rune Labs and Emerald Innovations in Parkinson's disease clinical trial
  • Novartis Tafinlar + Mekinist approved by FDA for pediatric patients with BRAF V600E low-grade glioma
  • AstraZeneca launches call for entries to the 2023 global R&D Postdoctoral Challenge
  • Pfizer invests $43 billion to battle cancer

Research & Development

  • 'Biohybrid' device could restore function in paralysed limbs
  • Scientists use tardigrade proteins for human health breakthrough
  • DNA treatment could delay paralysis that strikes nearly all patients with ALS
  • Scientists reveal a potential new approach to treating liver cancer
  • Normalizing tumor blood vessels may improve immunotherapy against brain cancer
  • Engineered bacteria find tumors, then alert the authorities
  • First nasal monoclonal antibody treatment for COVID-19 shows promise for treating virus, other diseases

Conferences & Events

  • SAE Media Group proudly presents the 4th Annual AI in Drug Discovery Conference
  • SAE Media Group's 6th annual 3D Cell Culture Conference
  • CPHI Frankfurt returns to pre-pandemic strength as pharma industry booms again
  • 14th Annual RNA Therapeutics: Investigating the next generation of genetic medicine through RNA based therapies
  • CPHI Excellence in Pharma Award Winners 2022
  • CPHI Frankfurt Report predicts huge funding overhang to drive contract services growth
  • CPHI Frankfurt 2022: Global pharma confidence hits record high in the annual CPHI Pharma Index

Regulatory Affairs

  • FDA grants Accelerated Approval for Alzheimer's disease treatment
  • FDA approves new HIV drug for adults with limited treatment options
  • FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer
  • FDA approves first gene therapy to treat adults with Hemophilia B
  • FDA approves first treatment for Acid Sphingomyelinase Deficiency, a rare genetic disease
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