Page 12 - Gene Therapy Program News Today

ALISO VIEJO, Calif.--(BUSINESS WIRE)--Apr 29, 2021-- The international rare disease patient advocacy organization, Global Genes, is pleased to announce the 6th Annual RARE Drug Development Symposium (RDDS) in partnership with the Orphan Disease Center of the University of Pennsylvania. The June 9-11 virtual event will connect and educate hundreds of advocates, clinicians, and academic and industry researchers to explore the latest science, opportunities, and challenges to the advancement of the...

Press release content from PR Newswire. The AP news staff was not involved in its creation. Aruvant Announces Oral Data Presentation at American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting April 27, 2021 GMT (PRNewsfoto/Aruvant Sciences) NEW YORK and BASEL, Switzerland, April 27, 2021 /PRNewswire/ -- Aruvant Sciences (“Aruvant”), a private company focused on developing gene therapies for rare diseases, today announced that data on ARU-1801, a potentially curative gene therapy...

Taysha Gene Therapies Inc (NASDAQ: TSHA) Regeneron Pharmaceuticals Inc (NASDAQ: REGN)Moderna Inc Moderna is an mRNA vaccine company that has been one of the biggest winners in the healthcare sector. The company is one of the first in the world to receive the Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for its coronavirus vaccine. In addition, its mRNA science is also contributing to the development of new drugs in the foreseeable future. For example, Moder...

- Urgency for effective treatment underscored by European Commission designation for investigational gene therapy PBKR03 - Global Phase 1/2 trial – GALax-C – PBKR03 planned to initiate in first half of 2021 PHILADELPHIA, April 05, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the European Commission has grant...

Press release content from Globe Newswire. The AP news staff was not involved in its creation. Scout Bio to Present at the BofA Securities 2021 Animal Health Summit Scout BioFebruary 18, 2021 GMT PHILADELPHIA, Feb. 18, 2021 (GLOBE NEWSWIRE) -- Scout Bio, a biotechnology company revolutionizing pet medicine by delivering a pipeline of one-time gene therapies for major chronic pet health conditions, today announced that Mark Heffernan, Scout Bio’s Chief Executive Officer, will present at the u...

Press release content from Globe Newswire. The AP news staff was not involved in its creation. Passage Bio Appoints Maxine Gowen, Ph.D. to Board of Directors Passage BioFebruary 19, 2021 GMT PHILADELPHIA, Feb. 19, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced the appointment of Maxine Gowen, Ph.D., to its board of directors, effectiv...

Fred Hutch, Univ. of Washington biotech spinout Ensoma launches with $70M in funding February 11, 2021 at 9:15 am Dr. Hans-Peter Kiem of the Fred Hutchinson Cancer Research Center. (Fred Hutch Photo) New spinout: Boston-based biotech startup Ensoma launched Thursday and announced a $70 million Series A funding round. The company is built on technology developed over two decades by Seattle researchers Dr. Hans-Peter Kiem of the Fred Hutchinson Cancer Research Center and Dr. André Lieber of the ...

Press release content from Globe Newswire. The AP news staff was not involved in its creation. FDA Clears IND Application for Passage Bio’s Gene Therapy Candidate PBKR03 for Treatment of ... Passage BioFebruary 8, 2021 GMT - Phase 1/2 trial expected to commence in first half of 2021  - Company has three INDs cleared for rare monogenic CNS disorders PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformativ...

Amicus Optimized Transgene Show Greater Substrate Reduction than Wild Type Construct Across All Tissues and Doses Further Validates Combining Amicus-Engineered Transgenes with Penn’s AAV Gene Therapy Technologies to Develop Next Generation Gene Therapies PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice. The results ...

New gene-based vaccine strategy receives grant from the Bill & Melinda Gates Foundation The AAVCOVID vaccine program, a novel gene-based vaccine strategy that utilizes an adeno-associated virus (AAV) vector, was granted an award for up to $2.1 million from the Bill & Melinda Gates Foundation. The grant will aid the effort to bring further preclinical validation to the AAV vaccine platform. An AAVCOVID vaccine candidate is set to enter clinical trials in 2021. The AAVCOVID vaccine program was fo...