On November 4, 2026 Ichnos Glenmark Innovation, Inc. (IGI), a global clinical-stage biotechnology company developing Multispecific therapeutics in oncology, reported that updated clinical data from the Phase 1 study of ISB 2001 (ABBV-2001) in relapsed/refractory multiple myeloma (RRMM) will be presented as an oral presentation at the 68th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

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"With longer follow-up from dose escalation and initial results from dose expansion, ISB 2001 (ABBV-2001) continues to demonstrate robust efficacy and tolerability in heavily pretreated patients with relapsed/refractory multiple myeloma," said Lida Pacaud, M.D., President and Chief Executive Officer of IGI. "These results represent an important milestone in the development of this first and only trispecific T-cell engager targeting CD38 x BCMA x CD3. Together with our partner, AbbVie, we remain focused on advancing ISB 2001 (ABBV-2001) as a potential new treatment option for patients with relapsed/refractory multiple myeloma (RRMM)."

ISB 2001 (ABBV-2001) Oral Presentation (abstract link):
First- and only-in-class CD38 x BCMA x CD3 trispecific T-cell engager ISB 2001 (ABBV-2001): Updated Phase 1 (TRIgnite-1) study data demonstrate robust efficacy and tolerability in relapsed/refractory multiple myeloma (RRMM)

Presenter: Binod Dhakal, M.D., Medical College of Wisconsin (MCW)
Date & Time: Saturday, December 12, 2026, 4–4:15pm CST
Room: La Nouvelle C at the Ernest N. Morial Convention Center, New Orleans
Session: 654. Multiple Myeloma: Pharmacologic Therapies: The March of Immunotherapy

About ISB 2001 (ABBV-2001)
ISB 2001 (ABBV-2001) is an investigational first- and only-in-class trispecific T-cell engager targeting CD38 and BCMA on myeloma cells and CD3 on T cells that is in development for the treatment of relapsed/refractory multiple myeloma (RRMM). Engineered using IGI`s BEAT Multispecific Platform, ISB 2001 (ABBV-2001) coordinates dual-antigen tumor recognition with immune engagement through T cells within a single molecular architecture. IGI and AbbVie formed an exclusive global licensing agreement for the trispecific in July 2025. ISB 2001 (ABBV-2001) is being evaluated in multiple myeloma through the Phase 1 TRIgnite-1 study and a Phase 1/2 combination study.

(Press release, Ichnos Sciences, NOV 4, 2026, View Source;utm_medium=rss&utm_campaign=ichnos-glenmark-innovation-igi-to-present-updated-clinical-data-for-isb-2001-abbv-2001-in-relapsed-refractory-multiple-myeloma-at-68th-ash-annual-meeting [SID1234674168])

On November 4, 2026 Geron Corporation (Nasdaq: GERN), a hematology-focused commercial stage biopharmaceutical company pursuing more meaningful moments for people living with blood cancer, reported financial results for the third quarter of 2026 and recent business highlights.

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"Another quarter of strong RYTELO demand growth reinforces our confidence in the LR-MDS commercial opportunity and our ability to reach more second-line patients. Increased demand growth is being driven by the community practices which, in our view, indicates the potential for RYTELO to have a long growth runway in the U.S.," said Harout Semerjian, President and Chief Executive Officer of Geron. "Outside the U.S. there is a clear opportunity for us to serve more patients and create additional value by broadening RYTELO’s access. As such, we have initiated a gated commercial launch to bring RYTELO to targeted EU markets with availability in Germany expected by the end of the year. Expanding access to therapies, along with pursing opportunistic innovation, is a key part of building a leading hematology company."

Recent Business Highlights

•
Reported RYTELO net product revenue of $60.1 million in the third quarter of 2026.

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Grew RYTELO demand by 10% in the third quarter 2026, compared to the second quarter 2026.

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Increased ordering accounts by roughly 8% in the third quarter 2026 to approximately 1,700.

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Initiated a gated commercial launch to bring RYTELO to eligible LR-MDS patients in targeted European markets. RYTELO is expected to be available in Germany later this year and France in 2027.

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Exhibited three posters at the Society of Hematologic Oncology (SOHO) 2026 Annual Meeting, including real-world evidence of imetelstat for the treatment of LR-MDS that was generally consistent with the Phase 3 IMerge trial and an updated post-hoc analysis of the Phase 2 IMbark study of imetelstat for the treatment of relapsed/refractory myelofibrosis.1-3

•
Delivered an oral presentation of retrospective real-world evidence of imetelstat in LR-MDS consistent with the IMerge trial along with a poster featuring a post-hoc study from the IMerge trial at the 88th Annual Meeting of the Japanese Society of Hematology (JSH).4-5

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Provided an oral presentation at the 2026 Annual Meeting of the German, Austrian and Swiss Societies for Hematology and Medical Oncology (DGHO, OeGHO, SGMO, SGH) highlighting post-hoc pooled analysis from the IMerge population that suggests transient cytopenias observed early in treatment may be the result of an on-target effect of imetelstat and may be predictive of a better therapeutic response in LR-MDS patients.6

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Nominated for the 2026 Prix Galien USA Award for Best Product for Rare/Orphan Diseases for RYTELO in LR-MDS, an award recognizing extraordinary scientific achievements in transforming health and improving lives.

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Published two manuscripts in peer-reviewed journals highlighting imetelstat’s role in LR-MDS and other hematological diseases:

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"Overall Survival and Long-Term Outcomes from the Randomized, Double-Blind, Placebo-Controlled, Phase III IMerge Trial of Imetelstat for Lower-Risk Myelodysplastic Syndromes" in Haematologica.7

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"Combining the Telomerase Inhibitor Imetelstat with the BCL-2 Inhibitor Venetoclax Enhances Apoptosis and Improves Survival in Acute Myeloid Leukemia Cellular and Animal Models" in the Journal of Clinical Medicine.8

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Announced the discontinuation of the Phase 3 IMpactMF trial evaluating imetelstat in JAK inhibitor R/R myelofibrosis based on a recommendation from the independent Data Monitoring Committee (DMC) that the trial was unlikely to meet the primary endpoint of overall survival. There were no new safety signals observed in the trial that change the known safety profile of imetelstat.

Third Quarter 2026 Financial Results

Cash and Marketable Securities

As of September 30, 2026, Geron had approximately $339.4 million in cash, cash equivalents, restricted cash and marketable securities, compared to $326.9 million as of June 30, 2026, which provides the Company with cash to fund its operating requirements for the foreseeable future.

Net Income (Loss)

For the three months ended September 30, 2026, the Company reported a net income of $1.4 million, or $0.00 per share, compared to a net loss of $18.4 million, or $0.03 per share, for the three months ended September 30, 2025. The change in net income (loss) is primarily attributable to increased revenue and continued financial discipline lowering operating expenses across the company.

Revenues

Total product revenue, net for the three months ended September 30, 2026, was $60.1 million, compared to $47.2 million for the three months ended September 30, 2025.

Costs and Operating Expenses

Total costs and operating expenses for the three months ended September 30, 2026, were $54.4 million, compared to $61.1 million for the three months ended September 30, 2025. The decrease is primarily due to lower personnel-related and clinical development expenses, partially offset by non-cash inventory-related expenses.

Cost of goods sold was approximately $6.1 million for the three months ended September 30, 2026, compared to $1.0 million for the three months ended September 30, 2025, which consisted of costs to manufacture and distribute RYTELO. The increase is primarily due to non-cash inventory-related expenses.

Research and development expenses for the three months ended September 30, 2026, were $12.7 million, compared to $21.1 million for the same period in 2025. The decrease in research and development expenses was primarily a result of lower personnel-related expenses and lower clinical and chemistry, manufacturing, and controls (CMC) costs.

Selling, general and administrative expenses for the three months ended September 30, 2026, were $35.7 million, compared to $39.0 million for the same period in 2025. The decrease was primarily due to lower personnel-related expenses.

2026 Financial Guidance

For fiscal year 2026, the Company continues to expect RYTELO net product revenue to be at the mid-to-high end of the $220 million to $240 million range. Geron now expects total operating expenses to be between $220 million and $230 million, down from its prior guidance range of $230 million to $240 million. Total operating expenses include non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, depreciation, and amortization.

Based on current operating plans and assumptions, the Company believes that its existing cash, cash equivalents, restricted cash and marketable securities, together with anticipated net revenues from U.S. sales of RYTELO, will be sufficient to fund projected operating requirements for the foreseeable future.

Conference Call

Geron will host a conference call at 8:00 a.m. ET on Wednesday, November 4, 2026, to discuss business updates and third quarter 2026 financial results.

A live webcast of the conference call will be available through the Investors section of the Company’s website at www.geron.com. A replay of the webcast will be archived and available on the Company’s website.

1.
Data presented at the Society of Hematologic Oncology (SOHO) 2026 Annual Meeting: Thomas J, et al. "Early Real-World Use and Hematologic Outcomes With Imetelstat in Patients With Myelodysplastic Syndromes: Insights From a United States Community Oncology Chart Review." Poster #MDS-775. September 9-12, 2026; Houston, TX, USA & Online

2.
Data presented at the Society of Hematologic Oncology (SOHO) 2026 Annual Meeting: Kuykendall AT, et al. "Updated Analysis of Overall Survival With Imetelstat in Relapsed or Refractory Myelofibrosis from IMbark Versus Real-World Data, and Assessment of Real-World Treatment Patterns: Updated Post Hoc Analysis With Extended Follow Up." Poster #MPN-269. September 9-12, 2026; Houston, TX, USA & Online

3.
Data presented at the Society of Hematologic Oncology (SOHO) 2026 Annual Meeting: Mascarenhas JO, et al. "Correlation Between IL-8 and TNF-Alpha and Overall Survival in Imetelstat-Treated Patients With Myelofibrosis Relapsed or Refractory to a Janus-Associated Kinase Inhibitor in the IMbark Trial." Poster #MPN-298. September 9-12, 2026; Houston, TX, USA & Online

4.
Data presented at the Annual Meeting of the Japanese Society of Hematology: Eid J, et al. "Real-World Experience With Imetelstat in Lower-Risk Myelodysplastic Syndromes: Insights From United States Claims Data." Oral Presentation #O1-11C-4. October 9-11, 2026; Kyoto, Japan

5.
Data presented at the Annual Meeting of the Japanese Society of Hematology: Eid J, et al. "Durable Clinical Outcomes With Imetelstat in Korean Patients With Lower-Risk-Myelodysplastic Syndromes." Poster #P3-12-5. October 9-11, 2026; Kyoto, Japan

6.
Data presented at the Annual Meeting of the German, Austrian and Swiss Societies for Hematology and Medical Oncology: Brauer D, et al. "Telomerase Inhibition With Imetelstat in Patients With Lower-Risk Myelodysplastic Syndromes: Integrating Molecular Findings, Cytopenias, and Clinical Outcomes." Oral Presentation #V755. October 9-12, 2026; Vienna, Austria

7.
Santini V, Komrokji RS, Savona MR, et al. Overall survival and long-term outcomes from the randomized, double-blind, placebo-controlled, phase III IMerge trial of imetelstat for lower-risk myelodysplastic syndromes. Haematologica. 2026 Sep 3. doi: 10.3324/haematol.2026.301456. Epub ahead of print. PMID: 42687814.

8.
Huang F, Rusbuldt JJ, Luistro L, Colton H, Eid J. Combining the Telomerase Inhibitor Imetelstat with the BCL-2 Inhibitor Venetoclax Enhances Apoptosis and Improves Survival in Acute Myeloid Leukemia Cellular and Animal Models. J Clin Med. 2026 Sep 23;15(19):7399. doi: 10.3390/jcm15197399. PMID: 42881859; PMCID: PMC13665851.

About RYTELO (imetelstat)

RYTELO (imetelstat) is an oligonucleotide telomerase inhibitor approved in the U.S. for the treatment of adult patients with lower-risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.

In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration and the European Commission.

Please see RYTELO (imetelstat) full Prescribing Information, including Medication Guide, available at View Source

About Geron Geron is a hematology-focused biopharmaceutical company pursuing more meaningful moments for people living with blood cancer. Because patients cannot wait, we approach our work with urgency, bringing together deep expertise in telomerase biology with patient perspectives and insights from clinical research and real-world care. Our medicine, RYTELO (imetelstat), is approved in the United States and European Union for certain patients with lower-risk myelodysplastic syndromes (LR-MDS). Geron is a commercial-stage company with offices in Parsippany, New Jersey and Foster City, California. To learn more, visit www.geron.com or follow us on LinkedIn.

(Press release, Geron, NOV 4, 2026, View Source [SID1234674167])

On November 4, 2026 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage biotechnology company developing gene therapies designed to restore tumor-suppressor function in cancer and insulin-producing function in diabetes, reported new research identifying TROP2 expression as a potential predictive biomarker of response to its lead drug candidate, Reqorsa (quaratusugene ozeplasmid), a gene therapy product, will be presented at the 2026 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics, taking place November 18-20, 2026 in Barcelona, Spain.

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The research collaborators found that low TROP2 expression was consistently associated with resistance to REQORSA across multiple preclinical models of non-small cell lung cancer (NSCLC). This finding was also supported by analysis of pretreatment tumor biopsies from patients enrolled in a clinical trial evaluating REQORSA.

"Discovering an applicable biomarker of response is a meaningful step toward a more precise approach to treating lung cancer with REQORSA," said Ryan Confer, President and Chief Executive Officer of Genprex. "These data show that low TROP2 expression was associated with primary resistance across multiple preclinical models, and this was supported by analysis of patient biopsies from a clinical trial. If confirmed in further studies, TROP2 could not only help us enhance our clinical trials by recruiting and selecting more applicable patients, but, more importantly, benefit future patients by ultimately delivering potentially more effective, personalized treatment options. This work reflects our commitment to a precision-medicine approach to treating lung cancer."

The TUSC2 tumor suppressor protein expression is downregulated or absent in more than 80% of NSCLC cases and nearly all small cell lung cancers. Previous studies have shown that REQORSA has antitumor activity, including in models resistant to targeted therapies and in immunotherapy-resistant KRAS/LKB1-mutant NSCLC. REQORSA’s effects are mediated by inhibiting MAPK and mTOR signaling, inducing cell-cycle arrest and apoptosis, and activating antitumor immune responses.

Key findings from the research include:

TROP2 expression associated with resistance: Approximately 50% of 10 NSCLC cell lines and 50% of 12 patient derived organoid models evaluated showed primary resistance to TUSC2 gene delivery. Protein-expression analyses consistently identified low TROP2 expression as a marker associated with resistance across multiple preclinical models.
Supporting clinical evidence: Analysis of pretreatment tumor biopsies from patients enrolled in a TUSC2 gene therapy clinical trial supported the association between TROP2 expression and treatment response.
TROP2 modulation influenced treatment sensitivity: Experimental manipulation of TROP2 expression altered TUSC2-induced cancer cell death, suggesting that TROP2-dependent signaling may play a role in therapeutic response. Further research is underway to understand these context-dependent effects.
Collectively, the findings identify TROP2 as a potential predictive biomarker for response to REQORSA and suggest that modulating TROP2 signaling may be a novel strategy to enhance the therapeutic efficacy of REQORSA in NSCLC. While these findings provide preclinical and supportive clinical evidence, additional research and prospective clinical evaluation will be required to establish TROP2’s predictive utility in patients receiving REQORSA.

The featured Genprex-supported poster is to be presented at the 2026 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics:

Title: TROP2 Predicts Response to TUSC2 Gene Therapy (Quaratusugene Ozeplasmid) and Its Modulation Sensitizes Non-Small Cell Lung Cancer to Treatment

Conference: 2026 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics

Topic: Drug Resistance and Modifiers

Session Date and Time: Wednesday, Nov. 18, 2026

Time: 12:00–6:00 p.m. CET

Abstract Number: 108

Poster Board Number: PB-108

Location: CCIB, Barcelona, Spain

The EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) 2026 poster is expected to be available on Genprex’s website following the presentation, subject to applicable conference disclosure requirements.

About Reqorsa Gene Therapy

Reqorsa (quaratusugene ozeplasmid), a gene therapy product, consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles in a lipoplex form (the Company’s Oncoprex Delivery System), which has a positive charge. REQORSA is injected intravenously and specifically targets cancer cells. REQORSA is designed to deliver the functioning TUSC2 gene to negatively charged cancer cells while minimizing uptake by normal tissue. Laboratory studies conducted at MD Anderson show that the uptake of TUSC2 in tumor cells in vitro after REQORSA treatment was 10 to 33 times the uptake in normal cells.

(Press release, Genprex, NOV 4, 2026, View Source [SID1234674166])

On November 4, 2026 FORE Biotherapeutics, a registration stage company dedicated to developing targeted therapies to treat patients with cancer, reported two abstracts that have been selected for presentation at two prestigious oncology medical meetings. The first is an oral presentation highlighting long-term efficacy and safety data from a Phase 1/2a study evaluating plixorafenib in patients with BRAF V600 primary CNS tumors and will be presented at the upcoming Society for Neuro-Oncology (SNO) 2026 Annual Meeting taking place in Philadelphia November 12-15, 2026. The second is a poster presentation featuring new preclinical data supporting plixorafenib’s unique mechanism of action and its potential as a future treatment for BRAF-altered colorectal cancer (CRC) and will be presented at the 38th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium (ENA 2026) taking place in Barcelona November 18-20, 2026.

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Stacie Peacock Shepherd, M.D., Ph.D., Chief Medical Officer of Fore, commented: "The data being presented at SNO this year are exciting because they show that plixorafenib has sustained response and activity in patients with BRAF-altered CNS tumors, including treatment durations exceeding 4 years, with a manageable safety profile with no cumulative or late-onset toxicities, and without the class-associated adverse events commonly observed with first generation RAF inhibitors."

"BRAF V600 mutation occurs in approximately 8% of colorectal cancer and is a highly aggressive tumor with poor prognosis due to limited responses and intrinsic and acquired resistance. As a novel paradox and dimer breaker BRAF inhibitor, we believe plixorafenib’s unique mechanism of action has the potential to be the ideal combination partner to mitigate resistance in colorectal cancer. At ENA this year, we will be presenting new preclinical data that demonstrates single-agent plixorafenib activity following standard therapies, along with compelling synergistic efficacy in vitro when combined with pan-RAS inhibition or widely used standard-of-care agents, including in resistant cell lines. We look forward to sharing all of these data with the scientific and medical communities later this month," concluded Dr. Peacock.

Oral Presentation Details at SNO 2026:

Title: Long-term safety and efficacy of plixorafenib in patients with BRAF-altered CNS neoplasms from the phase 1/2a PLX120-03 study
Presenter: Macarena De La Fuente, M.D., Sylvester Comprehensive Cancer Center, University of Miami
Abstract Number: CTNI-09
Session: Clinical Trials Oral Abstracts Session I
Date and Time: Friday, November 13, 2026, 10:54 – 10:59 a.m. ET

Poster Presentation Details at ENA 2026:

Title: Determinants of response and rational combination strategies for plixorafenib (PLX8394/FORE8394) in models of BRAF-altered colorectal cancer
Lead Author: Jessica Jang, Ph.D., Director, Translational Science, Fore Biotherapeutics
Poster Number: PB-117
Poster Session: Molecular Targeted Agents
Date and Time: Thursday, November 19, 2026, 1:00 – 3:00 p.m. CET

About Plixorafenib

Plixorafenib is a novel BRAF inhibitor, with a unique mechanism of action that functions both as a dimer and paradox breaker, and that has demonstrated a differentiated and compelling monotherapy profile in clinical studies. Plixorafenib received Breakthrough Therapy Designation by the U.S. Food and Drug Administration in April 2026. In a previously conducted Phase 1/2 study in patients with MAPK inhibitor naïve BRAF V600 primary recurrent CNS tumors (n=9), plixorafenib monotherapy demonstrated an ORR of 67% and a clinical benefit rate of greater than 75%. In patients with V600 alterations who were MAPK inhibitor naïve, plixorafenib achieved a 42% response rate with prolonged duration of response (mDOR 17.8 months), with a clinical benefit rate of >70%. Plixorafenib also demonstrated a favorable safety and tolerability profile across tumor types, including relative to existing standard of care treatments for various BRAF altered tumors, with a discontinuation rate due to drug-related adverse events of less than 2%. Fore believes plixorafenib has the potential to overcome the limitations and tolerability challenges of currently available BRAF inhibitors, including high rates of rash, hemorrhage and growth effects in younger patients, through its unique mechanism of action targeting both BRAF Class I and Class II alterations, while avoiding the limitations of the earlier generation BRAF inhibitors that led to rapid recurrence of disease and the need for combination with a MEK inhibitor.

(Press release, Fore Biotherapeutics, NOV 4, 2026, View Source [SID1234674165])

On November 4, 2026 Tangram Therapeutics ("Tangram"), a company committed to unlocking transformative RNAi medicines for meaningful patient impact, reported that preclinical data from TGM-148, a novel GalOmic medicine for bleeding disorders, will be presented in an oral session at the 68th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place 12–15 December 2026 in New Orleans, Louisiana.

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TGM-148 silences Protein Z-dependent Protease Inhibitor (ZPI), a natural regulator of coagulation. It is designed to combine robust hemostatic efficacy with low thrombotic risk and infrequent administration (four or fewer subcutaneous doses a year) to substantially reduce the burden of prophylaxis. In preclinical proof-of-concept studies, TGM-148 demonstrated efficacy in four distinct genetic disease models, including a model of von Willebrand disease (VWD), the planned entry indication.

Tangram’s oral presentation at ASH (Free ASH Whitepaper) will highlight TGM-148’s unique, context-dependent mechanism of action. In non-human primate plasma depleted of factor VIII, modeling a deficiency that causes hemophilia A and is common in VWD, TGM-148 dose-dependently enhanced thrombin generation. In non-depleted plasma, TGM-148 had no effect on thrombin generation, consistent with ZPI’s adaptive role in regulating coagulation. In addition, TGM-148 was well tolerated up to the highest dose tested in a repeat-dose toxicology study in healthy non-human primates, supporting a wide therapeutic window. Together, these findings demonstrate TGM-148’s potential to restore hemostatic balance in people with bleeding disorders without increasing thrombotic risk.

"We are pleased to present data at ASH (Free ASH Whitepaper) showcasing TGM-148’s context-dependent mechanism of action, which was a key reason we chose to pursue this novel target. With this differentiated mechanism, and the prospect of quarterly or less frequent dosing, TGM-148 has the potential to offer effective, patient-friendly prophylaxis with a wide therapeutic window", said Alan Whitmore, BM BCh, FFPM, PhD, Chief Scientific Officer of Tangram.

"People living with bleeding disorders need more prophylactic options, and the data Tangram will be presenting highlight the potential of TGM-148 as a novel approach. I am excited to see it progress through clinical development to evaluate ZPI silencing as a viable mechanism for rebalancing hemostasis." added Robert F. Sidonio, Jr MD, MSc, Pediatric Hematologist at Hemophilia of Georgia Pediatric Center for Bleeding and Clotting Disorders, Emory University School of Medicine.

Presentation Details

Title: TGM-148, a Novel GalNAc-siRNA Targeting ZPI, Demonstrates Context-dependent Hemostatic Rebalancing in Cynomolgus Monkeys
Publication Number: 953
Session Name: 801. Gene Therapies: Beyond Transplant: The Expanding Role of Gene Therapy in Hematology
Session Date & Time: 14 December, 14:45-16:15
Location: MCCNO – 388-390

About TGM-148
TGM-148 is a first-on-target GalOmic GalNAc-conjugated small interfering RNA (GalNAc-siRNA) designed to selectively silence Protein Z-dependent Protease Inhibitor (ZPI) in the liver, where it is produced before circulating in the blood. Its context-dependent mechanism of action aims to enhance clotting in response to bleeding in those with bleeding disorders, while maintaining a favorable safety profile.

In preclinical studies, TGM-148 demonstrated efficacy in four genetic bleeding disorder models and was well tolerated at supratherapeutic doses in wildtype animals. Together, these findings support TGM-148’s wide therapeutic window and provide a strong rationale for its development across a broad range of bleeding disorders, with VWD selected as the first indication. By offering the potential for quarterly or less frequent subcutaneous administration, TGM-148 is also designed to lower the barriers to prophylaxis, supporting broader uptake, better outcomes, and improved quality of life for patients.

(Press release, e-Therapeutics, NOV 4, 2026, View Source [SID1234674164])