GENE THERAPY RARE DISEASES – Page 12 – REGENHEALTHSOLUTIONS (RHS)

PIER MARIA FORNASARI
2019-10-03

ARM and NAMCP Publish Recommendations to Increase Patient Access in Joint Study, “Roadmap for Navigating Cell and Gene Therapy Value Demonstration and Reimbursement in U.S. Managed Care”

The Alliance for Regenerative Medicine (ARM) and the National Association of Managed Care Physicians (NAMCP) Medical Directors Institute announced today the joint release of their recent study of medical director and manufacturer perspectives on value demonstration and reimbursement for cell- and gene-based regenerative and advanced therapies. The study, entitled “Roadmap for Navigating Cell and Gene

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PIER MARIA FORNASARI
2019-10-03

New Gene Therapy Approach Led to Sustained Benefits in Mouse Model of Hemophilia A, Study Reports

A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research. The study, “Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice,” appeared

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PIER MARIA FORNASARI
2019-10-02

NIH researchers create new viral vector for improved gene therapy in sickle cell disease

Source NIH ‘Forward-oriented’ design might boost treatment effectiveness and broaden use. Researchers at the National Institutes of Health have developed a new and improved viral vector—a virus-based vehicle that delivers therapeutic genes—for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times

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PIER MARIA FORNASARI
2019-10-02

Gene therapies require advanced capabilities to succeed after approval and only five areas in the USA offer all four FDA approved

Gene therapies are getting approved. But there are major challenges ahead. A new report has been published by PwC Health Research Institute. After the thrill of approval has worn off and press coverage has faded, producers of gene therapies will be left with the challenge of getting their products to patients and getting paid for

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PIER MARIA FORNASARI
2019-10-01

In the pipeline: Surge of cell and gene therapies likely in 2020

Source BioProcess International Over 20 cell and gene-based therapies are expected to be filed or to receive approval decisions over the next 18 months, according to the Alliance for Regenerative Medicine (ARM). Earlier this year, the US Food and Drug Administration (FDA) laid out its action plan to deal with the large upswing in the

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PIER MARIA FORNASARI
2019-10-01

Potential Solutions To Current Pricing Models For Cell And Gene Therapies

Source: Life Science Leader Headlines about high-priced cell and gene therapies specifically can cause sticker shock for the patient. Exhibit A: Novartis’ Zolgensma, the spinal muscular atrophy (SMA) gene therapy treatment, priced at $2.125 million, is the world’s most expensive drug to date. But cell and gene therapies are their own animals; the differences between

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PIER MARIA FORNASARI
2019-10-01

New CRISPR-Cas9 variant may boost precision in gene editing

Researchers have developed a new variant of the gene editing technique CRISPR-Cas9 that has the potential to increase precision during gene therapy in humans. The new variant reduced unintended changes in DNA compared to its wildtype, suggesting it could play a role in gene therapies that require high precision. The study by researchers from Karolinska

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PIER MARIA FORNASARI
2019-09-24

ICER’s Leader: How to Assess the Value of Cell and Gene Therapies

Science is producing a growing number of gene and cell therapies to treat spinal muscular atrophy, leukemia and other conditions, but payers and providers don’t have the information they need to determine what they should pay for these treatments. That’s according to Steven Pearson, MD, founder and president of Boston-based Institute for Clinical and Economic

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PIER MARIA FORNASARI
2019-09-24

Novartis in political row after denying Belgian toddler compassionate access to gene therapy

The controversy over Novartis’ ultra-pricey gene therapy Zolgensma has intensified after it allegedly refused to supply the one-off therapy to a sick toddler in Belgium on compassionate grounds. According to The Brussels Times, the family of the toddler named Pia, who suffers from the ultra-rare disease spinal muscular atrophy (SMA) have been running a crowdfunding campaign

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PIER MARIA FORNASARI
2019-09-23

Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning

Source https://www.nature.com/articles/s41467-019-12281-8#Sec14 Highly specific Cas9 nucleases derived from SpCas9 are valuable tools for genome editing, but their wide applications are hampered by a lack of knowledge governing guide RNA (gRNA) activity. Here, we perform a genome-scale screen to measure gRNA activity for two highly specific SpCas9 variants (eSpCas9(1.1) and SpCas9-HF1) and wild-type SpCas9 (WT-SpCas9) in

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Category GENE THERAPY RARE DISEASES

ARM and NAMCP Publish Recommendations to Increase Patient Access in Joint Study, “Roadmap for Navigating Cell and Gene Therapy Value Demonstration and Reimbursement in U.S. Managed Care”

New Gene Therapy Approach Led to Sustained Benefits in Mouse Model of Hemophilia A, Study Reports

NIH researchers create new viral vector for improved gene therapy in sickle cell disease

Gene therapies require advanced capabilities to succeed after approval and only five areas in the USA offer all four FDA approved

In the pipeline: Surge of cell and gene therapies likely in 2020

Potential Solutions To Current Pricing Models For Cell And Gene Therapies

New CRISPR-Cas9 variant may boost precision in gene editing

ICER’s Leader: How to Assess the Value of Cell and Gene Therapies

Novartis in political row after denying Belgian toddler compassionate access to gene therapy

Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning