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Richmond congratulates Beam Therapeutics on the preliminary results from the first three dose levels of its ongoing Phase 1/2 study investigating a potential new therapy for Alpha-1 Antitrypsin Deficiency (AATD).
As a partner in this important research, Richmond is honoured to support work that has seen nine patients receive in-vivo base editing therapy to correct a disease-causing mutation. This marks a significant advancement in the development of gene-editing approaches for rare diseases, and Richmond looks forward to contributing to consecutive parts of this clinical trial.
Dr Ulrike Lorch, Chief Medical Director at Richmond Pharmacology, emphasised the significance of this milestone:
“This is an important step forward for patients with AATD and for applying in-vivo base editing to correct gene mutations that cause disease. Everyone involved in this collaborative effort, and most importantly the patients who came forward for this clinical trial, bring hope to people with a previously incurable disease.”
For full details, see the official Beam Therapeutics announcement.
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