Hematology Today—January 26, 2026

A focused weekly roundup of hematology and cell-therapy developments, covering regulatory designations, clinical trial activity, evolving endpoint policy, market signals, and a new therapy launch.

In Today’s Newsletter

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🧬 Wugen’s Sofi-cel earns FDA Breakthrough Therapy Designation [1] [US • 21 Jan 2026]

https://wugen.com/u-s-fda-grants-to-wugens-wu-cart-007-breakthrough-therapy-designation-for-treatment-of-relapsed-or-refractory-t-cell-acute-lymphoblastic-leukemia-t-cell-lymphoblastic-lymphoma/
Context: Allogeneic anti-CD7 CAR-T using CRISPR deletions of CD7 and TRAC; prior RMAT, Fast Track, Orphan, Rare Pediatric Disease, PRIME. Pivotal Phase 2 T-RRex is single-arm.
Key point: FDA granted Breakthrough Therapy Designation for Sofi-cel in R/R T-ALL/LBL based on Phase 1/2 data (endpoint not specified).
Implication: Introduces competition that may affect pricing and formulary access.

🧒 CHLA site opens pediatric access to off-the-shelf CD7 CAR-T trial [2] [US • 23 Jan 2026]

https://www.newswise.com/articles/trial-tests-novel-car-t-cell-therapy-for-children-with-t-cell-leukemia-at-children-s-hospital-los-angeles
Context: Phase 2 WU-CART-007 enrolling ≥1 year olds initially with ≥5% marrow blasts; MRD cohort planned after first 30 participants. Prior Phase 1/2 reported composite CR of 73% (source-reported).
Key point: CHLA joins pivotal program to test donor-derived, gene-edited CD7 CAR-T designed to avoid fratricide and mitigate GVHD risk.
Implication: May expand screening, initiation, and follow-up at scale.

📊 FDA draft: MRD-negativity and CR as surrogate endpoints in myeloma [3] [US • 22 Jan 2026]

https://www.americanpharmaceuticalreview.com/1315-News/623757-FDA-Draft-Guidance-Backs-MRD-and-Complete-Response-as-Surrogate-Endpoints-to-Speed-Multiple-Myeloma-Drug-Approvals/
Context: Draft guidance favors randomized trials, validated MRD assays, prespecified stats, and concurrent confirmatory studies; single-arm may be acceptable in some settings.
Key point: MRD negativity and CR could support accelerated approvals in MM if durability and follow-up are adequate.
Implication: May influence prescriber choice and payer reviews pending full data.

💉 Daratumumab 2025 net sales reported at USD 14.351B [4] [EU • 21 Jan 2026]

https://ir.genmab.com/news-releases/news-release-details/genmab-announces-net-sales-darzalexr-daratumumab-2025
Context: USD 8.266B US, USD 6.085B ROW; Genmab receives royalties under J&J license.
Key point: Continued revenue scale underscores entrenched position across MM treatment lines.
Implication: Introduces competition that may affect pricing and formulary access.

🧠 Arcellx to spotlight anito-cel’s D-Domain and treatment sequencing at Tandem [5] [US • 21 Jan 2026]

https://ir.arcellx.com/news/news-details/2026/Arcellx-to-Advance-the-Conversation-on-Its-Platform-and-the-Importance-of-Early-CAR-T-Access-for-Patients-with-Multiple-Myeloma-During-the-2026-Tandem-Meetings/default.aspx
Context: Presentations Feb 4–7, 2026, include fast off-rate D-Domain rationale, a sequencing simulation favoring CAR-T before bispecifics in 4L+ RRMM, and access inequity mapping.
Key point: Company positions anito-cel’s pharmacology and sequencing strategy while highlighting access gaps.
Implication: Signals pipeline investment and modality expansion.

🧪 Opna Bio’s zavabresib gets FDA Orphan Drug Designation in MF [6] [US • 21 Jan 2026]

https://www.opnabio.com/opna-bio-announces-orphan-drug-designation-granted-to-opn-2853-zavabresib-for-the-treatment-of-myelofibrosis/
Context: BET inhibitor evaluated with ruxolitinib in Phase 1 PROMise; ASH 2025 update noted spleen length reductions (n not stated in headline summary).
Key point: ODD granted for myelofibrosis; INN “zavabresib” assigned.
Implication: Signals pipeline investment and modality expansion.

🩸 Ouro’s BCMA×CD3 gamgertamig earns FDA Fast Track in AIHA and ITP [7] [US • 20 Jan 2026]

https://www.ouromedicines.com/news/012026/
Context: Open-label basket study in autoimmune cytopenias (NCT07083960) with SC dosing; separate basket in Sjögren’s and IIM (NCT07229144).
Key point: Fast Track designations support expedited development in AIHA and ITP, on top of prior ODDs.
Implication: May influence prescriber choice and payer reviews pending full data.

🛡️ Takeda launches low-IgA GAMMAGARD LIQUID ERC in the US [8] [US • 22 Jan 2026]

https://www.takeda.com/en-us/newsroom/news-releases/launch-gammagard-liquid-erc-us/
Context: Ready-to-use IG 10% with ≤2 µg/mL IgA for PI patients 2+ years, IV or SC; storage up to 24 months at room temp or 36 months refrigerated.
Key point: Only liquid IG in US with low IgA at this threshold per company, complementing GAMMAGARD S/D (planned discontinuation after Dec 2027 as inventory allows).
Implication: Introduces competition that may affect pricing and formulary access.

Why it matters

CD7 allogeneic CAR-T advances could reshape salvage options in aggressive T-cell leukemias.
FDA’s draft on MRD/CR may accelerate myeloma development and shift trial designs toward deeper response metrics.
Daratumumab’s scale sets a high commercial bar for late-line MM entrants.
Sequencing insights and access data from Arcellx can influence referral patterns and health-system planning.
Low-IgA IG availability may broaden options for PI patients with IgA sensitivity concerns.

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FAQ

What is Sofi-cel and why is the designation important? [1]

Sofi-cel is an allogeneic, CD7-targeted CAR-T for R/R T-ALL/LBL using CRISPR to remove CD7 and TRAC. FDA Breakthrough status enables intensive guidance and potential rolling review, aiming to speed development.

How is CHLA’s trial enrollment structured for WU-CART-007? [2]

The Phase 2 trial enrolls patients aged 1+ with ≥5% marrow blasts initially, with a planned expansion to an MRD cohort after the first 30 participants, reflecting interest in earlier disease states.

How would the FDA draft guidance change myeloma trials? [3]

It proposes MRD negativity and CR as surrogate endpoints for accelerated approval, emphasizing validated assays, randomized designs when possible, and concurrent confirmatory trials to verify clinical benefit.

What do daratumumab sales imply for competitors? [4]

With USD 14.351B in 2025 net sales, daratumumab remains a dominant MM backbone, implying new entrants must show clear differentiation in outcomes, convenience, or cost.

What is anito-cel’s D-Domain and the proposed sequencing takeaway? [5]

Anito-cel uses a compact D-Domain binder with a fast off-rate, which may enable effective tumor clearance without prolonged inflammation. A simulation model supports CAR-T before bispecifics in 4L+ RRMM, though this is modeled rather than clinical outcomes.

What does ODD mean for zavabresib in MF? [6]

ODD provides incentives like fee waivers and potential exclusivity if approved. Early data in combination with ruxolitinib are encouraging, but larger, controlled studies are needed.

Entities / Keywords

Wugen; Soficabtagene geleucel; Sofi-cel; WU-CART-007; CD7 CAR-T; T-ALL; T-LBL; CHLA; MRD; FDA draft guidance; multiple myeloma; MRD negativity; complete response; Genmab; Johnson & Johnson; daratumumab; DARZALEX; Arcellx; anitocabtagene autoleucel; anito-cel; D-Domain; BCMA; sequencing; access inequity; Opna Bio; OPN-2853; zavabresib; BET inhibitor; myelofibrosis; Ouro Medicines; gamgertamig; OM336; BCMA×CD3; AIHA; ITP; Takeda; GAMMAGARD LIQUID ERC; low-IgA IG; primary immunodeficiency.