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Journal Articles

BEAM-201 for the Treatment of Relapsed and/or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL): Initial Data from the Phase (Ph) 1/2 Dose-Exploration, Dose-Expansion, Safety, and Efficacy Study of Multiplex Base-Edited Allogeneic Anti-CD7 CAR-T-Cells

Caroline Diorio, Paul Shaughnessy, Nosha Farhadfar, Anjali S. Advani, Wen-Kai Weng, Hagop Youssoufian, Guo Chen, Felicia Nguyen Brown, Bahru Habtermariam, Lalit Kumar, Jigar Patel, Shangbin (Gary) Liu, Sunita Goyal, Amy Simon, Alex C. Minella, David T. Teachey
Journal: Blood
Blood (2024) 144 (Supplement 1): 4838.
https://doi.org/10.1182/blood-2024-203546
Published: 2024
Journal Articles

Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial Data from the BEACON Phase 1/2 Study of Autologous CD34 + Base Edited Hematopoietic Stem Cells in Sickle Cell Disease

Priya S. Chockalingam, Ling Lin, Guo Chen, Alex C. Minella, Yinzhong Chen, Vivien Sheehan, Nan Zhang, Myriam Armant, Aliya U. Zaidi, Robert Goodrich, Patrick C. Hines, Sunita Goyal, Amy Simon
Journal: Blood
Blood (2024) 144 (Supplement 1): 4957.
https://doi.org/10.1182/blood-2024-200875
Published: 2024
Journal Articles

Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

Ashish O. Gupta, Akshay Sharma, Haydar Frangoul, Jignesh Dalal, Julie Kanter, Asif Alavi, John DiPersio, Mary Eapen, Jennifer J. Jaroscak, Ernesto Ayala, Edward D. Ziga, Stacey Rifkin-Zenenberg, Alex C. Minella, Guo Chen, Yinzhong Chen, Priya S. Chockalingam, Ling Lin, Marcelyne Joseney-Antoine, Leanne Ianniello, Beth Gardner, Adam J. Hartigan, Giuseppe Ciaramella, Sunita Goyal, Amy Simon, Alexis Thompson, Matthew M. Heeney
Journal: Blood
Blood (2024) 144 (Supplement 1): 513.
https://doi.org/10.1182/blood-2024-204888
Published: 2024
Journal Articles

Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the Envision Open Label Extension

David J. Kuter, MD, Sioban Keel, MD, Charles J. Parker, MD, David C. Rees, MD, Ulrich Stölzel, MD, Paolo Ventura, MD, Manisha Balwani, Laurent Gouya, MD, PhD, Amy Simon, MD, Shangbin Liu, PhD, John J Ko, PharmD,MSc, Sean Rhyee, MD, M.P.H, Samuel M. Silver, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 13.
https://doi.org/10.1182/blood-2020-134564
Published: 2020
Journal Articles

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Eliane Sardh, MD PhD, Laurent Gouya, MD, Joseph Bloomer, MD, Manisha Balwani, MD MS, Dwight Bissell, MD, David C Rees, MA, MBBS, FRCP, Ulrich Stolzel, MD, John D. Phillips, PhD, Raili Kauppinen, MD PhD, Janneke Langendonk, MD PhD, Robert J. Desnick, MD PhD, Jean-Charles Deybach, MD, Herbert L. Bonkovsky, MD, Charles J. Parker, MD, H Naik, MS, CGC, Michael Badminton, MB, ChB, PhD, Penny Stein, MD PhD, Elizabeth Minder, MD, Jerzy Windyga, Pavel Martasek, MD PhD, Maria Domenica Cappellini, MD, Paolo Ventura, MD, Pauline Harper, MD PhD, Sverre Sandberg, MD PhD, Aasne Aarsand, PhD, Felix Alegre, MD, Aneta Ivanova, MD PhD, Neila Talbi, MD, Stephanie Rock, PhD, Amy Chan, PhD, Chang Heok Soh, PhD, Katherine A. McCarthy, W Querbes, PhD, Craig Penz, MA, Amy Simon, MD, Karl Anderson, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 2211.
https://doi.org/10.1182/blood.V130.Suppl_1.2211.2211
Published: 2017
Journal Articles

Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of Aln-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria

Eliane Sardh, MD PhD, Pauline Harper, MD PhD, Nabil Al-Tawil, MD, Manisha Balwani, MD MS, Karl Anderson, MD, Joseph Bloomer, MD, D. Montgomery Bissel, MD, Robert J. Desnick, MD, Charles J. Parker, MD, John D. Phillips, PhD, Herbert L. Bonkovsky, MD, Craig Penz, MA, Amy Chan, PhD, Chang-Heok Soh, PhD, William Querbes, PhD, Amy Simon, MD, Penelope Stein, MD PhD, David C. Rees, FRCP, FRCPath, MA, MBBS
Journal: Blood
Blood (2016) 128 (22): 2318.
https://doi.org/10.1182/blood.V128.22.2318.2318
Published: 2016
Journal Articles

A Subcutaneously Administered RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Phase 1 Study Results in Healthy Volunteers and Patients with Hemophilia a or B

Benny Sorensen, MD PhD, Tim Mant, MD, Akin Akinc, PhD, Amy Simon, MD, Lauren Melton, Christian Lynam, Andrew Strahs, PhD, Alfica Sehgal, PhD, Renta Hutabarat, PhD, Prasoon Chaturvedi, PhD, Scott Barros, PhD, Akshay Vaishnaw, MD PhD, Aln-AT Investigators
Journal: Blood
Blood (2014) 124 (21): 693.
https://doi.org/10.1182/blood.V124.21.693.693
Published: 2014