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US to procure 3.2 million doses of Covid-19 vaccine from Novavax

Pharmaceutical Technology

A protein-based vaccine, NVX-CoV2373 is created from the genetic sequence of the SARS-CoV-2 virus’ initial strain. It is formulated with the company’s saponin-based Matrix-M and also comprises a purified protein antigen.

Vaccine 162
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Cell and gene therapies: why advanced medicines call for specialised logistics

Pharmaceutical Technology

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine.

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Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

Delveinsight

Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks. Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases.

Vaccine 98
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Capturing progress in haemophilia care

pharmaphorum

Haemophilia is a rare genetic bleeding disorder, occurring in one in every 5,000 (Haemophilia A) to 30,000 (Haemophilia B) male births, and is associated with an increased bleeding tendency due to a deficiency of blood clotting proteins, called coagulation factors, which are needed for the blood to clot.†. What is haemophilia?

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FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies

The Pharma Data

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. Progeria is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein, progerin. ABOUT ZOKINVY (LONAFARNIB). LIMITATIONS OF USE.

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REGN-COV2 Independent Data Monitoring Committee Recommends Holding Enrollment in Hospitalized Patients with High Oxygen Requirements and Continuing Enrollment in Patients with Low or No Oxygen Requirements

The Pharma Data

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

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Considerations for Neuroscience Trials with Intrathecal Delivery and Other Methods of Direct CNS Administration

XTalks

What About Small Proteins and Antibodies Reaching Extracellular CNS Targets? The return of cell therapy-iPSCs (induced pluripotent stem cells) and genetically altered delivery cells. Ancillary services are also needed, such as a pharmacy to store, prepare and deliver the IP and labs such as a CSF lab to prepare samples.”.

Trials 83