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The role of a specialty pharmacy in enhancing patient access to rare and gene therapies

Bio Pharma Dive

Four critical components in which the right specialty pharmacy partner can bring rare and gene therapy manufacturers the scale, expertise and capabilities needed to drive enhanced patient access.

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

Pharmaceutical Technology

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research. Cold chain logistics, meanwhile, have never been so important.

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US FDA approves Pfizer’s migraine nasal spray Zavzpret

Pharmaceutical Technology

Zavzpret is claimed to be the first and only calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray approved to treat migraine. The company expects to launch Zavzpret in pharmacies in July.

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STAT+: Pharmalittle: FDA approves a gene therapy for a rare blood disorder; pharmacy chains ordered to pay $650 million over opioid crisis

STAT News

The therapy, Zynteglo, is just the third gene therapy approved by the FDA, and the first to target a chronic blood disease. It uses an engineered virus to deliver a gene fix into the bone marrow of patients. Zynteglo will cost $2.8 million per patient, making it one of the most expensive medicines ever sold in the U.S.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Cell & Gene Therapy Product Development: How Can You Build a Successful Partnership with Your CRO’s Project Manager?

XTalks

Over the past few years, there has been a significant expansion in the cell and gene therapy landscape, with an increasing number of therapies entering clinical trials and receiving regulatory approvals. “I think if we all go into these relationships with this mindset, we’ll achieve great things together.”

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New UK intracellular drug delivery centre to support RNA development

Pharmaceutical Technology

Strathclyde Institute of Pharmacy and Biomedical Sciences head professor Yvonne Perrie said: “This centre will provide new capabilities for mRNA-based drug development and manufacture of cost-effective vaccines and therapeutics in the UK, boosting not only economic growth but also security of supply.”