Pipeline &
Publications
Pipeline
Only CAR-T in AL Amyloidosis. Lead Program: NXC-201, a sterically-optimized BCMA-targeting CAR-T for AL Amyloidosis and other serious diseases.
Indication
Relapsed/ Refractory AL Amyloidosis
Therapy
A sterically-optimized chimeric antigen receptor T (CAR-T) cell therapy in development for the treatment of Relapsed/Refractory AL Amyloidosis and autoimmune diseases.
An autologous CAR-T therapy (using a patients own cells) targeting BCMA which seeks out and destroys dysfunctional plasma cells that drive these diseases.
PRE-CLINICAL
PHASE 1
PHASE 2
US FDA and EU EC Orphan Drug Designation ODD
US FDA and EU EC Orphan Drug Designation ODD
Indication
Undisclosed Other Serious Diseases
Therapy
A sterically-optimized chimeric antigen receptor T (CAR-T) cell therapy in development for the treatment of Relapsed/Refractory AL Amyloidosis and autoimmune diseases.
An autologous CAR-T therapy (using a patients own cells) targeting BCMA which seeks out and destroys dysfunctional plasma cells that drive these diseases.
PRE-CLINICAL
PHASE 1
PHASE 2
IND Enabled
Indication
UNDISCLOSED
Therapy
Cell Therapy
PRE-CLINICAL
PHASE 1
PHASE 2
Publications
Heather Landau, Shahzad Raza, Aaron Rosenberg, Jeffrey Zonder, Raymond Comenzo, Vaishali Sanchorawala, Michaela Liedtke, Amandeep Godara, Michael Rosenzweig, Charlotte Hughes, Eugene Brailovski, Mehrdad Abedi, Sham Mailankody, Jae Park, David Marks, Sridevi Rajeeve, Jennifer Liu. Initial Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of a CAR-T (NXC-201) in Relapsed or Refractory (R/R) Light Chain (AL). ASH Annual Meeting. Orlando, FL. December 7, 2025.
Heather Landau, Shahzad Raza, Aaron Rosenberg, Raymond Comenzo, Amandeep Godara, Jeffrey Zonder, Vaishali Sanchorawala, Jonathan Kaufman, Al-Ola Abdallah, Michaela Liedtke, Binoy Yohannan, Larry Anderson, Michael Rosenzweig, Hamza Hashmi, Charlotte Hughes, Eugene Brailovski, Mehrdad Abedi, Sham Mailankody, Jae Park, David Marks, Sridevi Rajeeve, Jennifer Liu. First 23-Patient Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of CAR-T in R/R Light Chain (AL) Amyloidosis, NXC-201. ASTCT Annual Meeting. Salt Lake City, UT, February 2026
Heather Landau, Shahzad Raza, Aaron Rosenberg, Raymond Comenzo, Amandeep Godara, Jeffrey Zonder, Vaishali Sanchorawala, Jonathan Kaufman, Al-Ola Abdallah, Michaela Liedtke, Binoy Yohannan, Larry Anderson, Michael Rosenzweig, Hamza Hashmi, Charlotte Hughes, Eugene Brailovski, Mehrdad Abedi, Sham Mailankody, Jae Park, David Marks, Sridevi Rajeeve, Jennifer Liu. First 23-Patient Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of CAR-T in R/R Light Chain (AL) Amyloidosis, NXC-201. ASTCT Annual Meeting. Salt Lake City, UT, February 2026
Heather Landau, Shahzad Raza, Aaron Rosenberg, Jeffrey Zonder, Raymond Comenzo, Vaishali Sanchorawala, Michaela Liedtke, Amandeep Godara, Michael Rosenzweig, Charlotte Hughes, Eugene Brailovski, Mehrdad Abedi, Sham Mailankody, Jae Park, David Marks, Sridevi Rajeeve, Jennifer Liu. Initial Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of a CAR-T (NXC-201) in Relapsed or Refractory (R/R) Light Chain (AL). ASH Annual Meeting. Orlando, FL. December 7, 2025.
Heather J. Landau, MD, Charlotte Hughes, MD, Aaron Seth Rosenberg, MD, Mehrdad Abedi, MD, Shahzad Raza, MD, Jeffrey A. Zonder, MD, Eugene Brailovski, MD, Sergio Giralt, MD, Sham Mailankody, MBBS, Jae H. Park, MD, Miguel-Angel Perales, MD, Saad Z Usmani, MD, David Marks, MBBS, PhD, Raymond Comenzo, MD, Sridevi Rajeeve, MD, Jennifer Liu, MD. Initial Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of a CAR-T (NXC-201) in Relapsed or Refractory (R/R) Light Chain (AL). ASCO Annual Meeting. Chicago, IL, June 3, 2025.
Heather J. Landau, MD, Charlotte Hughes, MD, Aaron Seth Rosenberg, MD, Mehrdad Abedi, MD, Shahzad Raza, MD, Jeffrey A. Zonder, MD, Eugene Brailovski, MD, Sergio Giralt, MD, Sham Mailankody, MBBS, Jae H. Park, MD, Miguel-Angel Perales, MD, Saad Z Usmani, MD, David Marks, MBBS, PhD, Raymond Comenzo, MD, Sridevi Rajeeve, MD, Jennifer Liu, MD. Initial Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of a CAR-T (NXC-201) in Relapsed or Refractory (R/R) Light Chain (AL). ASCO Annual Meeting. Chicago, IL, June 3, 2025
E Lebel, N Asherie, M Assayag, S Elias, S Grisariu, B Avni, S Kfir-Erenfeld, T Dubnikov-Sharon, R Alexander-Shani, N Bessig, A Shehadeh, A Ishtay, S Pimienta, V Vaistein, E Zimran, M Pick, Y C. Cohen, I Avivi Mazza, C Cohen, P Stepensky, and M E Gatt. Efficacy and Safety of Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) for the Treatment of Relapsed and Refractory AL Amyloidosis. 66th ASH Annual Meeting and Exposition, San Diego, CA, December 9, 2024.
Eyal Lebel, Nathalie Asherie, Shlomit Kfir-Erenfeld, Batia Avni, MD, Shlomo Elias, Miri Assayag, Tali Dubnikov-Sharon, Marjorie Pick, Rivka Alexander-Shani, Nomi Bessig, Shlomit Herr, Alaa Shehadeh, Aseel Ishtay, Shelly Pimienta, Vladimir Vainstein, Eran Zimran, Yael Cohen, Irit Avivi, Cyrille Cohen, Polina Stepensky, and Moshe E. Gatt. Efficacy and Safety of Anti–B-Cell Maturation Antigen Chimeric Antigen Receptor T-Cell for the Treatment of Relapsed and Refractory AL Amyloidosis. Journal of Clinical Oncology. December 9, 2024.
Eyal Lebel, Nathalie Asherie, Miri Assayag, Shlomo Elias, MD, PhD, Sigal Grisariu, MD, Batia Avni, MD, Shlomit Kfir-Erenfeld, Tali Dubnikov-Sharon, Rivka Alexander-Shani, Nomi Bessig, Alaa Shehadeh, Aseel Ishtay, Shelly Pimienta, Vladimir Vaistein, Eran Zimran, Marjorie Pick, Yael C. Cohen, Irit Avivi Mazza, Cyrille Cohen, Polina Stepensky and Moshe E Gatt, MD. Efficacy and Safety of Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) for the Treatment of Relapsed and Refractory AL Amyloidosis. 66th ASH Annual Meeting and Exposition, San Diego, CA, December 9, 2024.
M. Assayag, N. Asherie, S. Kfir-Erenfeld, Eyal Lebel, S. Grisariu, B. Avni, T. Dubnikov-Sharon1, S. Elias, M. Gatt, P. Stepensky. Academic BCMA-CART Cells HBI0101 (NXC-201), a Promising Approach for the Treatment of LC Amyloidosis. American Society of Gene & Cell Therapy 27th Annual Meeting. May 10, 2024.
ISA – XIX International Symposium on Amyloidosis (International Society of Amyloidosis) Presentation
E. Lebel, S. Kfir Erenfeld, N. Asherie, S. Grisariu, B. Avni, S. Elias, M. Assayag, T. Dubnikov, N. Zalcman, M. Pick, E. Zimran, C. Cohen, P. Stepensky, M.E. Gatt. Efficacy and Safety of Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) (NXC-201) for the Treatment of Relapsed and Refractory AL amyloidosis. International Society of Amyloidosis. May, 2024.
Lebel, S. Kfir Erenfeld, N. Asherie, S. Grisariu, B. Avni, S. Elias, M. Assayag, T. Dubnikov, N. Zalcman, M. Pick, E. Zimran, Y. Cohen, I. Avivi, C. Cohen, P. Stepensky, M.E. Gatt. Feasibility of a Novel Academic Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis, 65th ASH Annual Meeting and Exposition, San Diego, CA. Dec 10 2023
Lebel, S. Kfir Erenfeld, N. Asherie, S. Grisariu, B. Avni, S. Elias, M. Assayag, T. Dubnikov, N. Zalcman, M. Pick, E. Zimran, Y. Cohen, I. Avivi, C. Cohen, P. Stepensky, M.E. Gatt. Feasibility of a Novel Academic Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis, 65th ASH Annual Meeting and Exposition, San Diego, CA. October 2023
Lebel, S. Kfir Erenfeld, N. Asherie, S. Grisariu, B. Avni, S. Elias, M. Assayag, T. Dubnikov, N. Zalcman, M. Pick, E. Zimran, Y. Cohen, I. Avivi, C. Cohen, P. Stepensky, M.E. Gatt. Feasibility of a Novel Academic Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis, International Myeloma Society 20th Annual Meeting. 2023 Sep 27-30.
ASGCT – American Society of Gene & Cell Therapy — 29th Annual Meeting – May 19, 2023
Asherie N., Kfir-Erenfeld S., Lebel E., Assayag M., Grisariu S., Avni B., Gatt ME, Stepensky, P. BCMA-Targeted CART (HBI0101), a Safe and Efficacious Novel Modality of Treatment for Light Chain Amyloidosis (AL) Patients. Late Breaking Abstract. ASGCT – American Society of Gene & Cell Therapy 29 th Annual Meeting. May 19, 2023.
Kfir-Erenfeld S, Asherie N, Grisariu S, Avni B, Zimran E, Assayag M, Sharon TD, Pick M, Lebel E, Shaulov A, Cohen YC, Avivi I, Cohen CJ, Stepensky P, Gatt ME. Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis. Clin Cancer Res. 2022 Dec 1;28(23):5156-5166. doi: 10.1158/1078-0432.CCR-22-0637. PMID: 36107221.
Eyal Lebel, Nathalie Asherie, Shlomit Kfir-Erenfeld, Shlomo Elias, MD, PhD, Sigal Grisariu, MD, Batia Avni, MD, Miri Assayag, Tali Dubnikov-Sharon, Rivka Alexander-Shani, Nomi Bessig, Alaa Shehadeh, Aseel Ishtay, Shelly Pimienta, Vladimir Vaistein, Eran Zimran, Marjorie Pick, Yael C. Cohen, Irit Avivi Mazza, Cyrille Cohen, Polina Stepensky and Moshe E Gatt, MD. 66th ASH Annual Meeting and Exposition, San Diego, CA, December 2024.
S. Kfir-Erenfeld, N. Asherie, E. Lebel, V. Vainstein, M. Assayag, S.T. Dubnikov, S. Grisariu, B. Avni, S. Elias, R. Alexander-Shani, N. Bessig, A. Shehadeh, A. Ishtay, V. Zelmanovich, E. Zimran, M. Pick, I. Roziner, R.S. Kenett, Y.C. Cohen, I. Avivi, C.J. Cohen, M.E.E. Gatt, P. Stepensky. Clinical evaluation and determinants of response to HBI0101 (BCMA CART) therapy in relapsed/refractory multiple myeloma. Blood Adv. 2024 May 20:bloodadvances.2024012967. doi: 10.1182/bloodadvances.2024012967. Epub ahead of print. PMID: 38768428.
Lebel, N. Asherie, S. Kfir Erenfeld, S. Grisariu, B. Avni, S. Elias, M. Assayag, T. Dubnikov, N. Zalcman, M. Pick, E. Zimran, A. Shaulov, Y. Cohen, I. Avivi, C. Cohen, P. Stepensky, M.E. Safety and efficacy of a locally produced novel anti-BCMA Chimeric antigen receptor T-cell (CART) (HBI0101) for the treatment of relapsed and refractory multiple myeloma. 65th ASH Annual Meeting and Exposition, San Diego, CA. Dec 11, 2023
Lebel, N. Asherie, S. Kfir Erenfeld, S. Grisariu, B. Avni, S. Elias, M. Assayag, T. Dubnikov, N. Zalcman, M. Pick, E. Zimran, A. Shaulov, Y. Cohen, I. Avivi, C. Cohen, P. Stepensky, M.E. Safety and efficacy of a locally produced novel anti-BCMA Chimeric antigen receptor T-cell (CART) (HBI0101) for the treatment of relapsed and refractory multiple myeloma. 65th ASH Annual Meeting and Exposition, San Diego, CA. October 2023
Lebel, N. Asherie, S. Kfir Erenfeld, S. Grisariu, B. Avni, S. Elias, M. Assayag, T. Dubnikov, N. Zalcman, M. Pick, E. Zimran, A. Shaulov, Y. Cohen, I. Avivi, C. Cohen, P. Stepensky, M.E. Gatt. Efficacy and Safety of a Locally Produced Novel Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) (HBI0101) for the Treatment of Relapsed and Refractory Multiple Myeloma, International Myeloma Society 20th Annual Meeting. Sep 27-30 2023.
Assayag , N. Asherie, S. Kfir Erenfeld, B. Avni, T. Dubnikov, N. Zalcman, E. Lebel, E. Zimran, A. Shaulov, M. Pick, Y. Cohen, I. Avivi, C. Cohen, M. Gatt, S. Grisariu, P. Stepensky. Point-of-care CART manufacture and delivery for the treatment of multiple myeloma and AL amyloidosis: the experience of Hadassah Medical Center. Poster Presentation, European Society for Blood and Marrow Transplantation 49th Annual Meeting. 2023 Apr 23-26.
Assayag M, Asherie N, Kfir-Erenfeld S, Avni B, Dubnikov T, Zalcman N, Lebel E, Zimran E, Shaulov A, Pick M, Cohen Y, Avivi I, Cohen C, Gatt ME, Grisariu S, Stepensky P. Point-of-care CART manufacture and delivery: Expanding access to CART therapy via local institutions, Hadassah Medical Center experience. Poster Presentation, European Society for Blood and Marrow Transplantation and European Hematology Association 5th European CAR T-cell Meeting. 2023 Feb 9-11.
Asherie N, Kfir-Erenfeld S, Avni B, Assayag M, Dubnikov T, Zalcman N, Lebel E, Zimran E, Shaulov A, Pick M, Cohen Y, Avivi I, Cohen C, Gatt ME, Grisariu S, Stepensky P. Development and manufacturing of novel locally produced anti-BCMA CART cells for the treatment of relapsed/refractory multiple myeloma: phase I clinical results. Haematologica. 2022 Oct 6. doi: 10.3324/haematol.2022.281628. Epub ahead of print. PMID: 36200421.
Editorial – Sjöstrand M, Sadelain M. Driving CARs to new places: locally produced BCMA CAR T cells to treat multiple myeloma. Haematologica. 2023. doi: 10.3324/haematol.2022.282053. Epub ahead of print. PMID: 36794501. (PDF link)
Harush O, Asherie N, Kfir-Erenfeld S, Adler G, Barliya T, Assayag M, Gatt ME, Stepensky P, Cohen CJ. Preclinical evaluation and structural optimization of anti-BCMA CAR to target multiple myeloma. Haematologica. 2022 Oct 1;107(10):2395-2407. doi: 10.3324/haematol.2021.280169. PMID: 35354252; PMCID: PMC9521250.