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Liminal BioSciences Announces Sale of Priority Review Voucher for USD105M

Liminal BioSciences Announces Sale of Priority Review Voucher for USD105M
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Recent Amendments to the FDA Laws Attempt to Clarify and Improve Existing Systems | Mintz - Health Care Viewpoints

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Although the Biden-Harris Administration that assumed control of the Executive Branch on January 20, 2021 immediately ordered a regulatory freeze of new or pending rules while the new administration gets its bearings (as reported by our colleagues in this post), several important changes to the laws enforced by the Food and Drug Administration (FDA) were recently enacted by Congress. As legislative actions, those changes are of course unaffected by President Biden’s regulatory freeze and so we thought worth a summary to ensure our readers are up to speed on the large amount of activity that occurred in the final weeks of the 116th Congress and the Trump Administration. ....

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Amendments to the FDA Laws try to Clarify and Improve Systems

Thursday, January 28, 2021
Although the Biden-Harris Administration that assumed control of the Executive Branch on January 20, 2021 immediately ordered a regulatory freeze of new or pending rules while the new administration gets its bearings, several important changes to the laws enforced by the Food and Drug Administration (FDA) were recently enacted by Congress. As legislative actions, those changes are of course unaffected by President Biden’s regulatory freeze and so we thought worth a summary to ensure our readers are up to speed on the large amount of activity that occurred in the final weeks of the 116th Congress and the Trump Administration. ....

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Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency

Published: Jan 19, 2021
DALLAS (BUSINESS WIRE) Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.
“There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease,” said RA Session II, President, Founder and CEO of Taysha. “We are encouraged by the early evidence of TSHA-105’s disease-modifying approach and believe these designations will help us potentially accelerate the development of this exciting program. We look forward to working with the FDA to make TSHA-105 available t ....

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FDA Grants Rare Pediatric Disease, Orphan Drug Designation to Taysha's Rare Epilepsy Candidate

Published: Jan 19, 2021
By Brandon May
The U.S.
grantedrare pediatric disease and orphan drug designations for Taysha Gene Therapies’ AAV9-based gene treatment candidate currently in development for epilepsy caused by SLC13A5 deficiency.
SLC13A5, a rate form of infantile epilepsy caused by SLC13A5 gene mutations, is an autosomal recessive disorder characterized by developmental delay as well as seizures that occur within the first few days of life.
The gene therapy company’s TSHA-105 candidate is the AAV9-based gene therapy granted these designations and is poised to be the first treatment for SLC13A5-related epilepsy. There are no currently approved treatments for epilepsy caused by SLC13A5, explained Taysha’s President, Founder and CEO, RA Session II, in a statement. “We are encouraged by the early evidence of TSHA-105’s disease-modifying approach and believe these designations will help us potentially accelerate the development of this ex ....

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