Orca Bio Presents New Data at the 2026 Tandem Meetings of ASTCT® and CIBMTR® Reinforcing Orca-T® as a Durable, High-Precision Cell Therapy for Hematological Malignancies

Press release

Menlo Park, CA

February 5, 2026

Orca Bio Presents New Data at the 2026 Tandem Meetings of ASTCT® and CIBMTR® Reinforcing Orca-T® as a Durable, High-Precision Cell Therapy for Hematological Malignancies 

New retrospective analysis showed Orca-T with reduced intensity conditioning (RIC) demonstrated favorable OS, RFS and GRFS compared to PTCy  
Company announced first patients dosed in the Phase 2 SERENE-T study evaluating Orca-T with RIC 
Orca-T demonstrated superior OS and RFS with reduced non-relapse mortality in patients with myelodysplastic syndromes compared to PTCy-based transplants  
New manufacturing and distribution report found 100% of Orca-T products were distributed within 70 hours vein-to-vein time with consistent purity  
Patients with haploidentical donors treated with Orca-Q® showed promising outcomes in survival, GVHD and relapse-free survival   

MENLO PARK, CA, Feb. 5, 2026 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced new clinical data presented at the 2026 Tandem Meetings of ASTCT® and CIBMTR® from February 4-7 in Salt Lake City. 

 

Orca-T with Reduced Intensity Conditioning  

“Patients undergoing reduced intensity conditioning allogeneic stem cell transplantation often face a tradeoff between tolerability and long-term disease control,” said Caspian Oliai, MD, medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center, and primary investigator on the SERENE-T Phase 2 study. “The clinical evidence being generated today, which suggests Orca-T may improve key outcomes by reducing GVHD without increasing infection risk or relapse rate, provides a strong foundation for our ongoing evaluation of Orca-T in this setting. The dosing of the first patients in the Phase 2 SERENE‑T study further strengthens this initial momentum, representing a meaningful step forward in expanding the investigation of Orca‑T for patients undergoing reduced intensity conditioning.” 

 

SERENE-T (NCT07216443) is a new multicenter, open-label Phase 2 trial evaluating the safety, tolerability and efficacy of Orca-T, Orca Bio’s lead investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) undergoing reduced intensity conditioning (RIC) or non-myeloablative conditioning (NMA). The first patients were treated this year at Vanderbilt University, UCLA and Oregon Health & Science University (OHSU) - Knight Cancer Institute. The study continues to enroll patients with plans to open at additional transplant centers across the U.S. 

 

A new analysis compared outcomes from the single-center, open-label Phase 1 investigator-sponsored trial evaluating Orca-T versus a historical cohort of patients from the CIBMTR registry who received a conventional allogeneic hematopoietic stem cell transplant (alloHSCT) with post-transplant cyclophosphamide (PTCy). All patients were aged 60-75 (median 68 years), had a 7/8 or 8/8 human leukocyte antigen (HLA)-matched donor, and were given a RIC for the treatment of AML, acute lymphoblastic leukemia (ALL), MDS or myeloproliferative neoplasm (MPN). 

 

Patients receiving Orca-T (n=53) compared with PTCy (n=587) demonstrated improved: 

  • Overall survival at one year (OS; 88% vs 72%) and two years (84% vs 61%) 
  • Relapse-free survival at one year (RFS; 82% vs 61%) and two years (79% vs 53%) 
  • Graft-versus-host-disease relapse-free survival at one year (GRFS; 72% vs 54%) and two years (72% vs 45%) 
  • Relapse rates at one and two years (9.7% vs 23%, 9.7% vs 30%) and non-relapse mortality at one and two years (NRM; 8% vs 16%, 12% vs 17%).  

At one year, rates of Grade 3-4 acute and moderate-to-severe chronic graft versus host disease (aGVHD, cGVHD) were 0% and 10% with Orca-T, respectively, compared to 6% and 10% with PTCy. At two years, rates of cGVHD were 10% with Orca-T and 12% with PTCy. 

 

Orca-T Versus PTCy in Patients with Myelodysplastic Syndromes 

 

A post-hoc analysis of patients aged 18-65 with MDS compared pooled results from the Phase 3 Precision-T study and the Phase 1b study of Orca-T to a historical cohort from the CIBMTR registry of patients who received a conventional alloHSCT and PTCy in the myeloablative conditioning (MAC) HLA-matched setting. Patients with MDS who were treated with Orca-T (n=25) demonstrated higher one-, two- and three-year OS of 100% compared to the PTCy cohort (n=95) with 80%, 70% and 62%, respectively. At one year, the Orca-T arm showed RFS of 95% versus 64% with PTCy and NRM of 0% versus 9.9%, respectively. Notably, these trends were observed across subgroups including age and donor type. 

 

A similar analysis was conducted across multiple hematologic malignancies, including MDS, AML and ALL with consistent results. In this post-hoc analysis, Orca-T (n=164) demonstrated higher OS compared to PTCy (n=380) at one, two and three years (94% vs 82%, 85% vs 73% and 82% vs 65%, respectively). At one year, RFS was 78% with Orca-T compared with 70% with PTCy, while NRM was 2.7% versus 7.7%, respectively. These findings were consistent with the results observed in a subgroup of patients over 50 years of age. 

 

Reliable Manufacturing and Nationwide Distribution of Orca-T 

 

A manufacturing and distribution analysis from the Phase 3 and Phase 1b Orca-T studies conducted between December 2019 and September 2024 reported on the production of 243 clinical cell therapies, including 215 from single-day and 28 from two-day collections. Overall, 100% of products were delivered to transplant centers across the U.S. within 70 hours, with 99% infused within 72 hours. Product quality was consistent across all three Orca-T components: hematopoietic stem cells, regulatory T-cells and conventional T-cells. 

 

These results demonstrate the feasibility of reliably manufacturing and distributing Orca-T at scale while maintaining high product purity within a controlled logistics framework, supporting multicenter clinical studies and potential future commercial application. 

 

“These data continue to reinforce the strength of Orca-T across both clinical outcomes and operational execution,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. “The consistency of these results, along with our ability to reliably manufacture and deliver Orca-T across the U.S., highlights the potential of this therapy to make a meaningful difference for patients with hematologic malignancies. As we move toward a potential launch later this year, we remain focused on executing with the same level of commitment and rigor to support patients, clinicians and transplant centers.” 

 

Orca-Q for Patients with Haploidentical Donors 

 

New findings from the ongoing Phase 1 study of Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy, evaluated 39 patients with AML, ALL or MDS and a haploidentical donor who received MAC with Bu/Flu/Thiotepa (BFT), TBI/Flu/Thio (TFT) or TBI/Flu. All patients engrafted by Day +19 (median 11 days) and demonstrated encouraging rates of OS at one, two and three years (80%, 77% and 77%, respectively). Patients demonstrated RFS of 77% and GRFS of 72%, with low incidences of Grade 3-4 aGVHD at Day +180 and moderate-to-severe cGVHD at one year (8.1% and 0%, respectively). Outcomes were further improved in the TFT subgroup (n=14) across OS (85%), RFS (85%), GRFS (85%), aGVHD (0%) and cGVHD (0%). 


About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment of multiple hematologic malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration (FDA). The Biologics License Application (BLA) for Orca-T is currently under Priority Review with the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.   

About Orca-Q

Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies, including in patients with haploidentical and mismatched donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform.  

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. The company’s manufacturing platform uses single-cell precision to create personalized cell therapy products intended to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy. For more information, visit www.orcabio.com.  

Trademarks or registered trademarks used in this press release are the property of their respective owners.  

Contact:

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Kelsey Grossman

media@orcabio.com

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Joshua Murray

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Orca Bio Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for Orca-T® to Treat Hematological Malignancies

Press release

Menlo Park, CA

October 6, 2025

Orca Bio Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for Orca-T® to Treat Hematological Malignancies 

FDA assigns PDUFA target action date of April 6, 2026 
If approved, Orca-T would be the first allogeneic T-cell immunotherapy for the treatment of hematological malignancies including acute myeloid leukemia, acute lymphoblastic leukemia and myelodysplastic syndromes  

MENLO PARK, CA, October 6, 2025 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) seeking approval for Orca-T, its lead investigational allogeneic T-cell immunotherapy, for the treatment of hematological malignancies including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and myelodysplastic syndromes (MDS). 

 

The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.

"A stem cell transplant has been the only potentially curative option for many people with AML, ALL or MDS, however treatment-related toxicities too often hinder patient recovery. Acceptance of the Orca-T BLA marks a pivotal moment in our ability to deliver a first-in-class therapy designed to improve survival free from complications like graft versus host disease,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. “Supported by positive Phase 3 clinical data, today’s regulatory milestone reflects important recognition of the transformative potential of Orca-T. We look forward to working collaboratively with the FDA on the review of our application with the goal of advancing Orca-T and making it available to patients in need.” 

The BLA submission for Orca-T is supported by positive results from the pivotal Phase 3 study, Precision-T (NCT04013685), a randomized, open-label multi-center study that evaluated the safety, efficacy and tolerability of Orca-T compared to conventional allogeneic hematopoietic stem cell transplant (alloHSCT) in patients with AML, ALL and MDS. The study met its primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) with Orca-T versus alloHSCT. 

About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated for the treatment of multiple hematological malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration. 

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. The company’s manufacturing platform uses single-cell precision to create proprietary, uniquely-defined products designed to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy. For more information, visit www.orcabio.com. 

Trademarks or registered trademarks used in this press release are the property of their respective owners. 

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

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Orca Bio Announces FDA Review Extension of BLA for Orca-T for the Treatment of Hematologic Malignancies

Press release

Menlo Park, CA

April 1, 2026

Orca Bio Announces FDA Review Extension of BLA for Orca-T for the Treatment of Hematologic Malignancies 

New PDUFA Target Action Date of July 6, 2026 allows FDA additional time to complete its review 

MENLO PARK, CA, April 1, 2026 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has extended the review timeline of its Biologics License Application (BLA) for Orca-T for the treatment of patients with hematologic malignancies. The new Prescription Drug User Fee Act (PDUFA) target action date is July 6, 2026. 

 

The extension follows Orca Bio's recent submission of updated chemistry, manufacturing and controls (CMC) information in response to requests from the FDA as part of the standard review process. The FDA classified the submission as a Major Amendment (MA) to the BLA, which extends the review by three months. Importantly, the FDA has not requested any additional clinical data. Orca Bio believes that the updated information submitted in the amendment does not affect the benefit-risk conclusions of the BLA. 

 

"We appreciate the frequent engagement with the FDA throughout the review process,” said Nate Fernhoff, co-founder and chief executive officer at Orca Bio. “Our continued focus is on preparing for the potential approval and commercial launch of Orca-T. We remain committed to working with the Agency, physicians and the broader blood cancer community to deliver this important therapy to patients with hematologic malignancies as quickly as possible.”  

 

The BLA for Orca-T was granted Priority Review by the FDA and Orca-T previously received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation. 

About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment of multiple hematologic malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified hematopoietic stem cells, regulatory T-cells and conventional T-cells derived from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration (FDA).  

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. The company’s manufacturing platform uses single-cell precision to create personalized cell therapy products intended to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy. For more information, visit www.orcabio.com.  

Trademarks or registered trademarks used in this press release are the property of their respective owners.  

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Visit our newsroom
Orca Bio Presents Positive Data Demonstrating the Potential for Orca-T and Orca-Q to Expand Treatment to Additional Patient Groups at the 65th ASH Annual Meeting

Press release

Menlo Park, CA

December 9, 2023

Orca Bio Presents Positive Data Demonstrating the Potential for Orca-T and Orca-Q to Expand Treatment to Additional Patient Groups at the 65th ASH Annual Meeting

Orca-T, Orca Bio’s lead investigational high-precision cell therapy, showed similar results in older patients undergoing myeloablative conditioning as seen in younger patients across disease control, non-relapse mortality and overall survival
Orca-T in older patients given a reduced intensity conditioning regimen demonstrated curative potential with no incidence of relapse and encouraging relapse-free survival
Orca-T showed positive results in patients with intermediate to high-risk myelodysplastic syndrome
Orca-Q, Orca Bio’s second investigational high-precision cell therapy, showed encouraging data in graft-versus-host-disease-free and relapse-free survival in patients with a haploidentical donor without the use of post-transplant cyclophosphamide 

MENLO PARK, CA, Dec. 9, 2023 – Orca Bio, a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders, today announced positive new data that suggest its investigational high-precision cell therapies, Orca-T and Orca-Q, have the potential to deliver improved clinical outcomes across different age ranges, donor types and conditioning regimens relative to existing standard of care allogeneic hematopoietic stem cell transplants (alloHSCT). The results presented at the 65th American Society of Hematology (ASH) Annual Meeting showed promising data with the use of Orca Bio’s cell therapies across several patient groups where there is significant unmet medical need.

Orca-T in Older Patients with Myeloablative Conditioning

Results highlighted in an oral presentation showed Orca-T’s ability to deliver similar outcomes in older patients undergoing myeloablative conditioning (MAC) as younger patients. Notably, Orca-T delivered similar results across disease control, non-relapse mortality (NRM) and overall survival (OS).

“While the use of MAC offers the best chance of a cure, it also increases life-threatening complications in older patients receiving standard of care alloHSCT,” said presenting author Caspian Oliai, M.D., medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center. “These latest findings suggest a cell therapy like Orca-T, which has the potential to offer a cure while reducing toxicity and lowering treatment-related mortality, may allow older patients to better tolerate MAC. This approach could expand curative treatment to older patients who might not be offered a MAC regimen today, and potentially provide physicians with an important new option in our ongoing efforts to balance the risk of relapse with transplant-related side effects.”

In a subgroup analysis, 64 patients from Orca Bio’s ongoing multi-center Phase 1b clinical trial who received Orca-T and busulfan, fludarabine and thiotepa (BFT) conditioning for the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), mixed phenotype leukemia, chronic myeloid leukemia (CML) and myelodysplastic syndromes (MDS) were divided into two age groups: 18-54 years of age (n=39), and 55 years of age and older (n=25). With a median follow-up of 12 months, the following outcomes were reported: 

  • Relapse-free survival (RFS) was 84.8% in younger patients and 82.3% in older patients.
  • NRM was 0% in both groups.
  • OS was 100% and 95.5% in the younger and older patient groups, respectively.
  • Events of grade 3-4 acute graft versus host disease (GvHD) and moderate-to-severe chronic GvHD were low across the younger group (0 and 3, respectively) and older group (1 and 3, respectively).

Across all patients, Orca-T continued to be manufactured reliably and delivered with vein-to-vein times of 72 hours or less across the U.S.

Orca-T in Older Patients with Reduced Intensity Conditioning 

Additional data from a single-center open-label Phase 1 clinical trial presented at ASH looked at the outcomes of older patients treated with Orca-T and a reduced intensity conditioning (RIC). These patients were found to be unfit for a MAC regimen due to the significant risks associated with it, particularly in older age. At 12 months, patients treated with a RIC Orca-T (n=15) saw no observable compromise in curative outcomes, with no patients experiencing relapse (0%). Patients also saw encouraging RFS (79%) and a low incidence of both grade 3-4 acute and chronic GvHD (0% and 7%, respectively).

Collectively, these findings support the potential for Orca-T to treat older patients with hematological malignancies, whether they are given a MAC or RIC regimen, with no new safety signals reported.

Orca-T in Patients with MDS

New data was also presented on the performance of Orca-T in patients with intermediate to high-risk MDS. Current treatments for MDS aren’t often curative, and many patients relapse or become resistant to first-line treatment. There remains an unmet need for new, more effective but tolerable strategies to manage MDS.

In this subgroup from the ongoing multi-center Phase 1b clinical trial, Orca-T demonstrated promising results in this patient population (n=16). At one year, RFS with Orca-T was 94%. No patients experienced grade 3-4 acute GvHD, and moderate-to-severe chronic GvHD occurred in two patients. The rates of NRM and OS with Orca-T were 0% and 94%, respectively.

Orca-T is currently being evaluated in a pivotal Phase 3 clinical study for the treatment of AML, ALL and MDS at leading transplant centers across the U.S.

Orca-Q for Patients with Haploidentical Donors

In an oral presentation, Orca Bio shared updated data from a multi-center Phase 1 clinical trial of its second high-precision cell therapy, Orca-Q, in patients with a haploidentical donor without the use of post-transplant cyclophosphamide (PTCy).

“Orca-Q is a first-in-class therapy that has the potential to improve patient outcomes and reduce the risk of graft versus host disease without the use of PTCy,” said presenting author Samer Srour, M.D., M.S., Department of Stem Cell Transplantation and Cellular Therapy at The University of Texas MD Anderson Cancer Center. “While PTCy has increased the use of haploidentical HSCTs, it can bring a myriad of risks and toxicities that we didn’t experience with Orca-Q. These updated results in an expanded group of patients continue to support Orca-Q’s promise to overcome the challenges of haploidentical stem cell transplant by providing a solution where patients and physicians may no longer have to compromise between the risk of relapse and the risk of GvHD.”

The positive outcomes from an expanded group of 33 patients with AML, ALL and CML support Orca-Q as a potential treatment option for patients with a haploidentical donor. With a median of 375 days follow-up, updated safety and efficacy data include:

  • No patients experienced moderate-to-severe chronic GvHD (0%). 
  • There was one event of grade 3 acute GvHD and no grade 4 acute GvHD. 
  • NRM was 9%.
  • RFS, GRFS and OS at one year were 82%.

Additionally, no new safety signals were identified. The estimated incidence of CTCAE grade 2 and greater than grade 3 infections at one year were 9% and 15%, respectively. 

“At Orca Bio, we understand the challenges providers face in achieving the right balance in offering blood cancer patients the best option for a cure with optimal quality of life,” said Ivan Dimov, Ph.D., co-founder and chief executive officer of Orca Bio. “We are pleased to present these latest findings at ASH that add to our growing body of clinical evidence which underscore the potential of our novel high-precision platform to expand potentially life-saving treatment to more patient groups who could benefit.”

The full ASH presentations will be made available on www.orcabio.com.

About Orca-T

Orca-T is an investigational high-precision allogeneic cell therapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T includes infusions containing regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading transplant centers across the U.S. and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

About Orca-Q

Orca-Q is an investigational high-precision allogeneic cell therapy being evaluated in clinical trials for the treatment of hematologic malignancies in patients with haploidentical donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform. Orca-Q has the potential to improve patient outcomes and reduce the risks of toxicities without the use of post-transplant cyclophosphamide (PTCy) in patients unable to identify a full human leukocyte antigen (HLA) match. 

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders. Our investigational products are designed to safely replace a patient’s diseased blood and immune system with a healthy one, delivering significantly better outcomes with dramatically fewer risks than the standard of care. Our manufacturing platform uses single-cell precision to create proprietary, uniquely-defined products that have the potential to transform allogeneic cell therapy. At Orca Bio, our mission is to make curative cell therapies both more effective and safer, and in doing so, push past the field’s current boundaries and redefine its future. For more information, visit www.orcabio.com and follow Orca Bio on Twitter: @OrcaBio.

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Visit our newsroom
Orca Bio to Present Positive Clinical Data on Orca-T at the 50th Annual Meeting of the EBMT

Press release

Menlo Park, CA

April 11, 2024

Orca Bio to Present Positive Clinical Data on Orca-T at the 50th Annual Meeting of the EBMT

Presentations include data from an expanded group of older patients treated with Orca Bio’s investigational high-precision cell therapy, Orca-T, and reduced intensity conditioning

MENLO PARK, CA, April 11, 2024 – Orca Bio, a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders, today announced new data will be presented during the 50th Annual Meeting of the EBMT in Glasgow, United Kingdom.

Specifically, results from a single-center open-label Phase 1 clinical trial of an expanded group of older patients (n=20) treated with Orca Bio’s lead investigational high-precision cell therapy, Orca-T, and a reduced intensity conditioning (RIC) regimen will be presented. Preliminary data with Orca-T and RIC in patients with hematologic malignancies showed encouraging rates of relapse-free survival (77%). With a median follow-up of 9.65 months, no patients have experienced relapse to date (0%), demonstrating the curative potential for Orca-T in this historically challenging to treat patient group. An earlier dataset evaluating Orca-T and RIC in this population was presented at the 2023 American Society of Hematology (ASH) Annual Meeting.

A second presentation will highlight findings from a non-randomized retrospective analysis comparing Orca-T plus single-agent tacrolimus with a standard of care allogeneic stem cell transplant (alloHSCT) plus post-transplant cyclophosphamide (PTCy)-based graft versus host disease (GvHD) prophylaxis in patients with hematologic malignancies receiving myeloablative conditioning (MAC). When compared to the historical PTCy cohort in this patient population, Orca-T offered potentially favorable rates of relapse-free survival, non-relapse mortality and overall survival. These data were previously presented at the 2024 Tandem Meetings, Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood and Marrow Transplant Research (CIBMTR). 

“At Orca Bio, we continue to build clinical evidence to support the profile of Orca-T and its potential to expand life-saving treatment to patients while also reducing treatment-related risks,” said Scott McClellan, M.D., Ph.D., chief medical officer at Orca Bio. “We look forward to joining top healthcare professionals from around the world at EBMT and sharing the latest findings that support the potential for Orca-T to offer a new curative solution for providers and their patients.”

The EBMT abstracts are available at https://www.ebmt.org/. 

Details of the Orca Bio presentations follow:

Title: Phase 1 Trial Results for Patients with Advanced Hematologic Malignancies Treated with Orca-T Cell Therapy with Reduced Intensity Conditioning and Single Agent Tacrolimus 

Poster Code: A121

Date and Time: Monday, April 15, 2024 at 18:00 BST

Location: Hall 3 North

Title: A Retrospective Analysis Comparing Orca-T to Post-Transplant Cyclophosphamide Based Allogeneic Hematopoietic Stem Cell Transplant in Patients with Matched Unrelated Donors Receiving Myeloablative Conditioning

Poster Code: A107

Date and Time: Monday, April 15, 2024 at 18:00 BST

Location: Hall 3 North


About Orca-T

Orca-T is an investigational high-precision allogeneic cell therapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T includes infusions containing regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading transplant centers across the U.S. and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders. Our investigational products are designed to safely replace a patient’s diseased blood and immune system with a healthy one, delivering significantly better outcomes with dramatically fewer risks than the standard of care. Our manufacturing platform uses single-cell precision to create proprietary, uniquely-defined products that have the potential to transform allogeneic cell therapy. At Orca Bio, our mission is to make curative cell therapies both more effective and safer, and in doing so, push past the field’s current boundaries and redefine its future. For more information, visit www.orcabio.com and follow Orca Bio on Twitter: @OrcaBio. 

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com


Visit our newsroom
Orca Bio to Present Positive Clinical Outcomes with Orca-T in Patients with AML at 2024 ASCO Annual Meeting

Press release

Menlo Park, CA

May 23, 2024

Orca Bio to Present Positive Clinical Outcomes with Orca-T in Patients with AML at 2024 ASCO Annual Meeting

Relapse-free survival and overall survival were 82.5% and 100% at 12 months, respectively, in 37 patients with AML, an aggressive form of blood cancer

Non-relapse mortality at 12 months was 0%

Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading treatment centers across the U.S.

MENLO PARK, CA, May 23, 2024 – Orca Bio, a biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced it will present new data at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois, from May 31–June 4. The presentation will highlight outcomes of its lead investigational allogeneic T-cell immunotherapy, Orca-T, in patients with acute myeloid leukemia (AML). 

AML is an aggressive form of blood cancer and the most common acute leukemia in adults. There are an estimated 20,800 new diagnoses and nearly 11,200 deaths in the U.S. each year.

“Today, only a fraction of adults diagnosed with AML undergo curative treatment with standard of care allogeneic hematopoietic stem cell transplant due to the serious and life-threatening risks often associated with it,” said Rawan Faramand, M.D., assistant member of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center. “These new findings suggest Orca-T has the potential to offer a cure with low rates of treatment-related mortality, and could provide an important option for patients with AML. With its goal of alleviating the tradeoff between the risk of relapse and the risk of toxicities observed with standard of care stem cell transplant, this novel approach has the potential to expand curative treatment to many more patients.”

A new subanalysis from the ongoing multi-center Phase 1b single-arm trial evaluated outcomes with Orca-T among a subgroup of 37 patients with AML who had a median age of 51 years and median follow-up of 14 months. All patients received myeloablative conditioning (MAC) with busulfan, fludarabine and thiotepa (BFT), followed by Orca-T and single-agent graft versus host disease (GvHD) prophylaxis with tacrolimus. At 12 months, results found relapse-free survival was 82.5% (95% CI: 65.0, 91.7) and non-relapse mortality was 0%. Overall survival was 100% (95% CI: 100, 100) at 12 months. The safety profile of Orca-T in this subgroup was consistent with the larger Phase 1b population with no new safety signals identified. Across all patients, Orca-T was manufactured reliably and delivered with vein-to-vein times of 72 hours or less across the U.S.

“We’re pleased to present at ASCO for the first time and share the latest findings that support the potential for Orca-T to offer a curative solution and fulfill a significant unmet need for patients with AML,” said Scott McClellan, M.D., Ph.D., chief medical officer at Orca Bio. “These data continue to advance our mission of expanding potentially life-saving treatment to patients and their providers who face limited viable options today.”

Details of the Orca Bio presentation follow:

Title: Treatment of Acute Myeloid Leukemia with Orca-T

Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

Abstract Number: 6552

Poster Code: 111

Date and Time: June 3, 2024, from 9AM - 12PM CDT

Location: Oncology Professionals Hall

The ASCO abstracts are available at https://conferences.asco.org/am/abstracts. 

About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T is comprised of highly purified regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in a pivotal Phase 3 clinical trial at leading transplant centers across the U.S. and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of cancer, autoimmune diseases and genetic blood disorders. Our investigational products are designed to safely replace a patient’s diseased blood and immune system with a healthy one, delivering significantly better outcomes with dramatically fewer risks than the standard of care. Our manufacturing platform uses single-cell precision to create proprietary, uniquely-defined products that have the potential to transform allogeneic cell therapy. At Orca Bio, our mission is to make curative cell therapies both more effective and safer, and in doing so, push past the field’s current boundaries and redefine its future. For more information, visit www.orcabio.com and follow Orca Bio on X: @OrcaBio. 

Contact:

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Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com


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Orca Bio Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Orca-Q® for the Treatment of High-Risk Hematologic Malignancies

Press release

Menlo Park, CA

April 28, 2026

Orca Bio Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Orca-Q® for the Treatment of High-Risk Hematologic Malignancies 

RMAT designation based on promising preliminary Phase 1 clinical data, including positive outcomes across overall survival, acute and chronic GVHD and non-relapse mortality 
Phase 1 trial was recently expanded to include additional cohorts of patients receiving reduced intensity or non-myeloablative conditioning with either matched, 7/8 mismatched or haploidentical donors  
New clinical findings and longer follow-up data expected in 2026 

MENLO PARK, CA, April 28, 2026 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy for the treatment of high-risk hematologic malignancies.

"The FDA’s RMAT designation for Orca-Q recognizes the significant unmet need for patients with serious hematologic malignancies and validates the promising clinical findings from our ongoing Phase 1 study,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. “As this is our second cell therapy candidate to receive RMAT status, we have experienced firsthand how this program can support accelerated development and enhanced communication with the FDA. With our newly expanded Phase 1 study continuing to enroll patients and additional data expected later this year, we remain focused on advancing our high-precision approach to deliver an important new treatment to patients with blood cancer.”  

 

The RMAT application included data from the ongoing Phase 1 clinical trial (NCT03802695) evaluating Orca-Q for the treatment of hematologic malignancies across six treatment cohorts, including in patients with haploidentical donors, and in patients receiving non-myeloablative or reduced intensity conditioning. Clinical findings demonstrated durability and encouraging outcomes across key metrics, including overall survival, acute and chronic graft versus host disease (GVHD) and non-relapse mortality. Enrollment is ongoing, with new data expected to be presented at upcoming scientific congresses. Additional details can be found on www.ClinicalTrials.gov.  

 

RMAT designation is a specialized FDA program created to accelerate the development and review of promising new therapies, including cell therapies, intended to treat serious or life-threatening conditions. To qualify, a therapy must demonstrate preliminary clinical evidence suggesting the potential to address unmet medical needs. This designation provides important opportunities during the drug development process, including increased FDA guidance and eligibility for priority and rolling reviews, as well as accelerated approval pathways. By streamlining these regulatory milestones, the program aims to bring transformative innovations to patients more quickly. 

About Orca-Q

Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies, including in patients with haploidentical and mismatched donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform.  

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. The company’s manufacturing platform uses single-cell precision to create personalized cell therapy products intended to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy. For more information, visit www.orcabio.com.  

Trademarks or registered trademarks used in this press release are the property of their respective owners.  

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

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Orca Bio to Present Clinical Data on Its Pipeline of High-Precision Cell Therapies at the 52nd Annual Meeting of the EBMT

Press release

Menlo Park, CA

March 2, 2026

Orca Bio to Present Clinical Data on Its Pipeline of High-Precision Cell Therapies at the 52nd Annual Meeting of the EBMT 

Patient reported outcomes with Orca-T® from the pivotal Precision-T Phase 3 study will be presented including quality of life and rates of rehospitalizations compared to conventional alloHSCT 
Data evaluating the use of Orca-T with reduced intensity conditioning versus PTCy for the treatment of hematologic malignancies will be shared  
Additional presentations will highlight the manufacturing and nationwide distribution data reported on Orca–T and clinical findings of Orca-Q® in patients with haploidentical donors 

MENLO PARK, CA, March 2, 2026 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced that clinical data will be presented in seven oral and poster sessions at the 52nd Annual Meeting of the EBMT from March 22-25 in Madrid. 

 

The encore presentations will include data on the company's pipeline of investigational allogeneic T-cell immunotherapies for the treatment of multiple hematologic malignancies, including Orca-T and Orca-Q. 

 

“Our participation at this year’s meeting of the EBMT provides an important opportunity to engage directly with the global transplant community as we continue our shared efforts to advance the field of allogeneic stem cell transplant,” said Nate Fernhoff, Ph.D., co-founder and chief executive officer of Orca Bio. “As we progress toward the potential FDA approval of Orca-T, the strength and consistency of our data reinforce our commitment to delivering a new therapy for patients with blood cancer. We look forward to being in Madrid to connect with clinicians and partners from around the world and to participate in scientific exchange focused on improving patient outcomes.” 

 

The EBMT abstracts are now available at www.ebmt.org/annual-meeting-2026. Details of the Orca Bio presentations follow: 

 

Oral Session: OS14 | Graft Manipulation and Conditioning   

Title: Orca-T Demonstrates Favorable Quality of Life and Healthcare Resource Use Compared to Standard AlloHSCT plus Tac/MTX for GVHD Prevention in a Randomized Phase 3 Clinical Trial (Precision-T) 

Date and Time: Wednesday, March 25 at 08:30 AM – 08:39 AM CET 

Location: N107 

 

Oral Session: OS14 | Graft Manipulation and Conditioning   

Title: Clinical Outcomes in Orca-T and Registry-Based Post-Transplant Cyclophosphamide Patients: An Observational Comparison 

Date and Time: Wednesday, March 25 at 08:39 AM – 08:48 AM CET 

Location: N107 

 

Oral Session: OS12 | Acute GVHD - Clinical 

Title: Cost-Effectiveness of Orca-T vs Allo-HCT with Conventional GVHD Prophylaxis for the Treatment of Advanced Hematologic Malignancies in the United States 

Date and Time: Tuesday, March 24 at 2:39 PM – 2:48 PM CET 

Location: N101-102 

 

Poster Session: A 

Title: Interim Clinical Outcomes in Orca-T with Reduced Intensity Conditioning: An Observational Comparison to Registry-Based Post-Transplant Cyclophosphamide Patients 

 Presentation ID: A038 

Date and Time: Monday, March 23 at 6:00 PM – 7:00 PM CET 

Location: Pavilion 9 

 

Poster Session: A 

Title: Scalable Manufacturing and Nationwide Distribution of Orca-T: A Precision-Engineered Allogeneic Immune Cell Therapy 

 Presentation ID: A145 

Date and Time: Monday, March 23 at 6:00 PM – 7:00 PM CET 

Location: Pavilion 9 

 

Poster Session: A 

Title: Preliminary Safety and Efficacy of Myeloablative Orca-Q in Patients with Haploidentical Donors 

 Presentation ID: A043 

Date and Time: Monday, March 23 at 6:00 PM – 7:00 PM CET 

Location: Pavilion 9 

 

Poster Session: A 

Title: Clinical Outcomes in Myelodysplastic Syndrome Patients Treated with Orca-T or Post-Transplant Cyclophosphamide Patients: A Registry-Based Comparison 

 Presentation ID: A085 

Date and Time: Monday, March 23 at 6:00 PM – 7:00 PM CET 

Location: Pavilion 9 

About Orca-T

Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment of multiple hematologic malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration (FDA). The Biologics License Application (BLA) for Orca-T is currently under Priority Review with the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.   

About Orca-Q

Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies, including in patients with haploidentical and mismatched donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform.  

About Orca Bio

Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases. The company’s manufacturing platform uses single-cell precision to create personalized cell therapy products intended to replace a patient’s diseased blood and immune system with a healthy one. At Orca Bio, we are on a mission to redefine what’s possible for patients by transforming the field of curative allogeneic cell therapy. For more information, visit www.orcabio.com.  

Trademarks or registered trademarks used in this press release are the property of their respective owners.  

Contact:

Corporate Communications

Kelsey Grossman

media@orcabio.com

Investor Relations

Joshua Murray

ir@orcabio.com

Visit our newsroom