This week’s hematology update highlights groundbreaking CAR-T and gene therapy data, radioconjugate developments, and regulatory milestones across multiple therapy areas. Key updates include CARsgen’s CT071 in multiple myeloma, Cellectar’s iopofosine I-131 for Waldenström macroglobulinemia, and CSL Behring’s HEMGENIX gene therapy progress in Canada.
💊 CARsgen’s GPRC5D CAR-T CT071 data published in The Lancet Haematology [1] [China • 08 Oct 2025]
https://www.carsgen.com/en/news/20251008/
Context: Investigator-initiated Phase 1 trial (NCT05838131) in 20 relapsed/refractory multiple myeloma patients.
Key point: 100% objective response rate (50% sCR, 20% VGPR, 30% PR) with no dose-limiting toxicities and no ≥Grade 3 CRS.
Implication: May influence prescriber choice and payer reviews pending full data.
🧬 Cellectar’s iopofosine I-131 gains EMA CMA eligibility for Waldenström macroglobulinemia [2] [EU • 06 Oct 2025]
https://www.cellectar.com/news-media/press-releases/detail/376/
Context: EMA Scientific Advice Working Party deemed CMA filing acceptable for post-BTKi refractory WM; submission expected early 2026.
Key point: PRIME-designated radioconjugate showed 83.6% ORR in Phase 2b CLOVER WaM.
Implication: May influence prescriber choice and payer reviews pending full data.
🌞 Soligenix reports safety milestone in Phase 3 HyBryte™ FLASH2 study for CTCL [3] [US • 07 Oct 2025]
https://ir.soligenix.com/2025-10-07-Soligenix-Achieves-Important-Safety-Milestone
Context: DMC review found no safety concerns in confirmatory Phase 3 study of synthetic hypericin in early-stage CTCL.
Key point: Enrollment on track; blinded interim efficacy analysis planned 1H 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 Orca Bio’s Orca-T BLA accepted for Priority Review by FDA [4] [US • 06 Oct 2025]
https://orcabio.com/orca-bio-announces-fda-acceptance-and-priority-review
Context: Allogeneic T-cell therapy for AML, ALL, MDS received Priority Review; PDUFA Apr 6 2026.
Key point: Phase 3 Precision-T met primary endpoint of improved survival free of moderate-to-severe cGvHD vs alloHSCT.
Implication: May influence prescriber choice and payer reviews pending full data.
🧩 Kernal Bio secures up to $48 million ARPA-H award for in vivo mRNA CAR-T KR-402 [5] [US • 07 Oct 2025]
https://www.businesswire.com/news/home/20251007266824/en/
Context: ARPA-H EMBODY program funds Kernal and partners (Stanford, Dana-Farber, Jackson Lab) to develop in vivo CAR-T for B-cell malignancies and autoimmune disease.
Key point: KR-402 uses cell-selective mRNA and targeted LNPs to program T cells in situ; goal is 100-fold cost reduction vs ex vivo CAR-T.
Implication: Signals pipeline investment and modality expansion.
💰 Karyopharm raises $100 million, extends cash runway to 2Q 2026 [6] [US • 08 Oct 2025]
https://investors.karyopharm.com/2025-10-08-Karyopharm-Announces-Strategic-Financing-Transactions
Context: Transactions include new term loans, convertible notes and private placement.
Key point: Supports Phase 3 SENTRY (myelofibrosis) and XPORT-EC-042 (endometrial cancer); SENTRY topline due Mar 2026.
Implication: Signals pipeline investment and modality expansion.
🧬 CSL Behring and pCPA reach LOI for public reimbursement of HEMGENIX® in Canada [7] [CA • 06 Oct 2025]
https://newsroom.csl.com/2025-10-06-CSL-Behring-Signs-Letter-of-Intent
Context: LOI covers public listing outside Quebec for one-time gene therapy HEMGENIX (etranacogene dezaparvovec) for hemophilia B.
Key point: Based on HOPE-B Phase 3 data showing mean FIX activity ~37% at 4 years and 94% off prophylaxis.
Implication: Introduces competition that may affect pricing and formulary access.
🩸 Cureus study profiles infections in hospitalized sickle-cell patients in India [8] [IN • 10 Oct 2025]
https://www.cureus.com/articles/412771-clinical-and-microbiological-profile
Context: 211-patient observational study (2018–2023) at central India tertiary center.
Key point: Respiratory infections most common (34.6%); mortality 6.6%; procalcitonin > 2 ng/mL predictive of poor outcomes.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧪 Terumo BCT and Hemex Health partner to expand Gazelle™ Hb testing access [9] [Global • 09 Oct 2025]
https://www.terumo.com/newsrelease/detail/20251010/6756
Context: Terumo made minority investment in Hemex Health and joined its board to accelerate sickle-cell and Hb variant diagnostics.
Key point: Collaboration covers clinical research, digital integration and regional pilots to scale testing in low-resource settings.
Implication: May expand screening, initiation and follow-up at scale.
🧭 U.S. backs Kernal Bio’s in vivo CAR-T via ARPA-H EMBODY program [10] [US • 07 Oct 2025]
https://www.fiercebiotech.com/biotech/us-government-bankrolls-48m-kernals-mrna-car-t
Context: Fierce Biotech confirmed federal funding for Kernal and academic partners to advance KR-402 for hematologic cancers and autoimmune disease.
Key point: Funds support clinical development and mRNA 2.0 platform expansion despite broader government cuts to mRNA projects.
Implication: Signals pipeline investment and modality expansion.
Why it matters
- Hematology pipeline momentum spans cell therapy, radioconjugates, and gene therapy in both malignant and rare disease settings.
- Early-phase CAR-T and mRNA platform data highlight rapid translation of new modalities into regulatory and funding channels.
- Public-reimbursement progress for HEMGENIX illustrates post-approval value negotiation for gene therapy at national scale.
- Academic and industry collaboration remains central to sickle-cell diagnosis and infection management efforts in India and Africa.
- Financing transactions (e.g. Karyopharm) underscore the link between capital access and Phase 3 readout continuity in oncology.
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FAQ
What did CARsgen report for CT071 in multiple myeloma? [1]
CARsgen announced 100% ORR in 20 relapsed/refractory multiple myeloma patients, with no ≥Grade 3 CRS and one Grade 3 ICANS; median DoR and PFS not reached.
When will Cellectar file for EMA CMA of iopofosine I-131? [2]
Following EMA advice, Cellectar plans submission in early 2026; potential EU approval and launch in 2027.
What is Orca Bio’s regulatory timeline for Orca-T? [4]
The FDA Priority Review sets an action date of 6 Apr 2026 for its BLA in AML, ALL and MDS.
How much funding did Kernal Bio receive and for what goal? [5][10]
Kernal Bio secured up to $48 million from ARPA-H to develop in vivo mRNA CAR-T KR-402 and expand its mRNA 2.0 platform with academic collaborators.
What step did CSL Behring and pCPA take for HEMGENIX in Canada? [7]
They signed an LOI enabling provinces to list HEMGENIX for public reimbursement, broadening access to gene therapy for hemophilia B.
What were the main findings of the Indian sickle-cell infection study? [8]
Respiratory infections predominated; antimicrobial resistance was high; procalcitonin > 2 ng/mL linked to mortality; study proposed a pilot risk score (AUC 0.78).
Entities / Keywords
CARsgen Therapeutics • CT071 • GPRC5D • multiple myeloma • Cellectar Biosciences • iopofosine I-131 • Waldenström macroglobulinemia • Soligenix • HyBryte (SGX301) • cutaneous T-cell lymphoma • Orca Bio • Orca-T • AML • ALL • MDS • Kernal Bio • ARPA-H • KR-402 • Karyopharm Therapeutics • selinexor • ruxolitinib • myelofibrosis • CSL Behring • HEMGENIX • hemophilia B • Cureus • sickle cell disease • Terumo Blood and Cell Technologies • Hemex Health • Gazelle™ • point-of-care diagnostics.
References
https://www.carsgen.com/en/news/20251008/
https://www.terumo.com/newsrelease/detail/20251010/6756
https://www.fiercebiotech.com/biotech/us-government-bankrolls-48m-kernals-mrna-car-t