Sickle Cell Disease – VJRegenMed

Increasing patient recruitment to rare disease trials

Simon Ng — Mon, 14 Feb 2022 17:40:21 +0000

Will Chou, MD, Aruvant, New York, NY, provides his insights on approaches to solve recruitment issues in clinical trials for rare diseases such as sickle cell disease (SCD). He emphasizes the need to gain the trust of patients, who may not necessarily be knowledgeable of every aspect of the therapy, as well as being transparent with everyone involved in the trials. This interview took place at Advanced Therapies Week 2022.

MOMENTUM: ARU-1801 in sickle cell disease

Simon Ng — Mon, 07 Feb 2022 17:06:07 +0000

Will Chou, MD, Aruvant, New York, NY, presents preliminary results from the Phase I/II MOMENTUM study (NCT02186418) of ARU-1801, a lentiviral gene therapy, in patients with sickle cell disease (SCD). 5 patients are currently enrolled and the investigational therapy has a promising efficacy and safety profile, with no vaso-occlusive episodes (VOE) or hospital stays reported in patients. Dr Chou additionally reports a durable response in patients, with therapeutic hemoglobin detected in patients 3 years after initial administration. This interview took place at Advanced Therapies Week 2022.

ARU-1801, a novel lentiviral gene therapy for SCD

Simon Ng — Thu, 03 Feb 2022 12:25:15 +0000

Current investigational therapies for sickle cell disease (SCD) require conditioning chemotherapy such as busulfan to administer the genetically-modified stem cells in the patient’s bone marrow. Will Chou, MD, Aruvant, New York, NY, provides an overview of ARU-1801, for patients with SCD. The lentiviral gene therapy uses fewer doses of an alternative chemotherapy, melphalan, which results in fewer side effects such as thrombocytopenia and veno-occlusive liver disease, as well as shorter hospital stays. This interview took place at Advanced Therapies Week 2022.

Addressing issues in conducting clinical trials for rare diseases

Simon Ng — Thu, 03 Feb 2022 12:25:09 +0000

Will Chou, MD, Aruvant, New York, NY, gives an overview of challenges associated with conducting clinical trials for rare and orphan diseases such as sickle cell disease (SCD) and hypophosphatasia. Due the nature of these diseases, finding an adequate number of patients can be a barrier and Dr Chou additionally highlights the need to collaborate with patients groups to educate and build rapport with them for successful clinical trial recruitment. This interview took place at Advanced Therapies Week 2022.

HSC gene editing: current approaches and remaining challenges

admin_vjr_user — Thu, 19 Aug 2021 11:17:13 +0000

Valentina Vavassori, PhD, IRCCS San Raffaele Scientific Institute & Vita-Salute San Raffaele University, Milan, Italy, provides an overview of the current landscape for hematopoietic stem cell (HSC) gene editing and describes key gene editing approaches used including the non-homologous end joining (NHEJ) and homology-directed repair (HDR) pathways. Key lines of ongoing research to advance HSC gene editing approaches include the improvement of HDR efficiency in HSCs, as well as the development of new tools, such as CRISPR-associated transposases, to circumvent toxicity associated with double-strand breaks. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

ISCT 2021 highlights: BMT, gene therapy & lymphodepletion

admin_vjr_user — Thu, 24 Jun 2021 17:15:35 +0000

Jaap Boelens, MD, PhD, Memorial Sloan Kettering Cancer Center, New York, NY, offers some highlights in stem cell engineering and bone marrow transplantation (BMT) at the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021. Dr Boelens describes a plenary session exploring the latest data and considerations in gene therapy and BMT for the treatment of hemoglobinopathies, as well as a international debate on the use of different transplant platforms. In addition, he discusses a plenary focused on lymphodepletion, its impact on the efficacy and toxicity of chimeric antigen receptor (CAR) T-cell therapy and data on the pharmacokinetics of these agents. This interview took place during the ISCT Annual Meeting 2021.