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

XTalks

Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

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The regulatory round-up: Eight key FDA decisions

Drug Discovery World

The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February.

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Overcoming diverging regulatory expectations to bring CGTs to market

Drug Discovery World

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

The Pharma Data

The treatment induced HPV-specific T-cell responses and was associated with increased levels of immune cell infiltration in the tumors and expression of genes associated with activation of the immune system. Key findings of the trial: . – .

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Sanofi continued its growth trajectory. Strong increase in Q1 2021 business EPS(1) at CER

The Pharma Data

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

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