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Lysosomal Disorders

10 Cell and Gene Therapies to Watch in 2023

With confidence building, numerous cell and gene therapies will likely go before the FDA and other global regulatory agencies this year, in addition to key data readouts.

CGTLive’s Weekly Rewind – January 20, 2023

Review top news and interview highlights from the week ending January 20, 2023.

Late-Onset Pompe Disease Gene Therapy Cleared to Continue Trial

The FDA has lifted a clinical hold placed in the summer of 2022 due to a mild but medically significant case of peripheral sensory neuropathy.