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

XTalks

“Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. Terence Eagleton, MB BS, Senior Medical Director at the global clinical research organization (CRO) Medpace.

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PharmaDrug Enters Definitive Agreement for Acquisition of Sairiyo Therapeutics Inc. Who Recently Received Orphan Drug Designation from FDA for Esophageal Cancer

The Pharma Data

Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Food and Drug Administration (“FDA”) approval. PharmaDrug Inc.

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Pfizer and Valneva Issue Update on Phase 3 Clinical Trial Evaluating Lyme Disease Vaccine Candidate VLA15

Pfizer

GCP is the international ethical and scientific quality standard for clinical trials that all clinical researchers need to follow. The clinical trial remains ongoing with other sites not operated by the third party, and Pfizer continues to enroll new participants at those sites.

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TG Therapeutics Announces Pricing of Upsized Public Offering of Common Stock – Dec 15, 2020

The Pharma Data

The Company anticipates using net proceeds from the offering to fund the continued development of ublituximab and umbralisib, the potential in-license, acquisition, development and commercialization of other pharmaceutical products, and for general corporate purposes. Morgan Securities LLC, Goldman Sachs & Co.

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Phase 2 FIGHT Trial Results Presented at ASCO GI Validate Importance of FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy as a Frontline Targeted Treatment for FGFR2b+ Gastric and GEJ Cancers

The Pharma Data

Associate Professor of Medicine at UCLA, Co-director of the Gastrointestinal Oncology Program and Director of Early Phase Clinical Research at the Jonsson Comprehensive Cancer Center. Bemarituzumab is being developed in gastric and GEJ cancer as a targeted therapy for tumors that overexpress FGFR2b. Wainberg, M.D.,

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Molecular Partners Announces Collaboration With Novartis to Develop Two DARPin(R) Therapies Designed for Potential Use Against COVID-19

The Pharma Data

Novartis has been granted an option to in-license global rights of MP0420 and MP0423 – multi-targeted direct-acting antiviral therapeutic candidates demonstrating potential efficacy against COVID-19. Upon option exercise, Novartis would be responsible for all further development and commercialization activities.

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Moderna Announces Primary Efficacy Analysis in Phase 3 COVE Study for Its COVID-19 Vaccine Candidate and Filing Today with U.S. FDA for Emergency Use Authorization

The Pharma Data

“I want to thank the thousands of participants in our Phase 1, Phase 2 and Phase 3 studies, as well as the staff at clinical trial sites who have been on the front lines of the fight against the virus. These medically high-risk groups represent 42% of the total participants in the Phase 3 COVE study. Department of Defense Contract No.