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Expert Interview

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Discussing Beam's recent BEACON and ESCAPE data for BEAM-101 in Sickle Cell Disease presented at ASH 2024

Ticker(s): BEAM

Who's the expert?

Institution: Penn State Health

  • Director of the Pediatric Sickle Cell Program at Penn State Health Children’s Hospital since 2017
  • Treats 150 - 200 patients with Alzheimer’s Disease
  • Research interest includes movement disorders, biomarkers, Parkinson’s disease, and Alzheimer’s disease.

Interview Questions
Q1.

BEAM-101 patients achieved significant fetal hemoglobin (HbF) induction of over 60% by Month 1. Could you elaborate on the clinical implications of this level of HbF induction for sickle cell patients and how it may influence disease symptoms or progression?

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Q2.

One patient in the BEACON trial passed away due to respiratory complications related to busulfan conditioning, though unrelated to BEAM-101 itself. How critical is it for patients and clinicians to consider conditioning risks in autologous stem cell transplants, and what alternatives might BEAM-101 offer?

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Q3.

The ESCAPE data showed encouraging HbF induction in non-human primates using antibody-based conditioning. Could this approach potentially eliminate the need for chemotherapy in sickle cell treatment, and how might it change patient outcomes or broaden treatment eligibility?

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Q4.

With BEAM-101 treatment, patients demonstrated reduced sickle hemoglobin and fewer sickled red blood cells, similar to the sickle cell trait. Can you discuss the potential impact of these findings on the long-term health and quality of life for sickle cell patients?

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Q5.

How do you anticipate Beam’s base editing approach might improve the overall treatment experience for sickle cell patients, particularly in terms of engraftment speed and long-term management of symptoms?

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Q6.

As Beam gathers data on BEAM-101 with more patients and extended follow-up, what are the most critical markers of success in your view? How could these indicators shape the future of BEAM-101 as a curative option?

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Q7.

While the ESCAPE model has shown promise in non-human primates, what challenges do you foresee in bringing this conditioning method into human clinical trials, and what might be the regulatory considerations?

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Q8.

Beam’s approach centers on precision and predictable editing with base editing technology. Could you share your perspective on how this technology might influence the treatment landscape for hemoglobinopathies and potentially other genetic blood disorders in the future?

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