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Beam Therapeutics has announced compelling updated clinical data from its ongoing Phase 1/2 Trial of BEAM-302 as a potential treatment for Alpha-1 Antitrypsin Deficiency (AATD). This latest data supports a move to a pivotal next stage of trials.
Responding to this announcement, Dr Ulrike Lorch, Chief Medical Director at Richmond Pharmacology, said:
“This is an important step forward for patients with Alpha-1 Antitrypsin Deficiency (AATD) and for applying gene editing to correct disease-causing mutations. Richmond is proud to have played a leading role in this study, being the first clinic to dose a patient with Beam-302 and to have dosed the largest cohort patients on this trial.
“What makes these results especially meaningful is that we are now seeing the possibility of treating the disease at its source—helping the body produce the right protein – rather than just treating the symptoms.
“We are deeply grateful to the patients who have taken part. Their involvement is vital in helping to move this research forward and bring new treatments closer for the whole Alpha-1 community. We are also grateful for the support and collaboration of leading specialists at centres of Alpha-1 expertise around the UK.
We would warmly encourage anyone who has Alpha-1, or thinks they might have it, to get in touch with us to find out more about testing, support and opportunities to take part in research.”
BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation designed to directly correct the underlying genetic mutation that causes the severe form of Alpha-1 Antitrypsin Deficiency through base editing. In 2020, Richmond Pharmacology made history by becoming the first clinic in the world to carry out gene editing using a single LNP infusion into the liver.
For the full Beam Therapeutics news release see here https://investors.beamtx.com/press-releases
To find out about taking part in Alpha-1 research, fill out the form here or contact us on 020 7042 5802 AATD@richmondpharmacology.com
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