Evidence map›Paper›PMID 38027064›Full record

ArticleMolecular therapy. Methods & clinical development2023

Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity.

John R Lydeard, Michelle I Lin, Huanying Gary Ge, Amanda Halfond, Shu Wang, Mark B Jones, Julia Etchin, Gabriella Angelini, Juliana Xavier-Ferrucio, Jessica Lisle and 7 more

Erratum issuedOpen access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
2.6field-weighted citation impact, top 10% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

12 citing papers in PubMed, 17 citations in OpenAlex.

  1. Trial
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  7. Article
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  10. Biomanufacturing in gene and cell therapy.Molecular therapy. Methods & clinical development · 2024
    Article
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

17 authors at 1 institution in 2 countries.

John R LydeardVor Biopharma, Cambridge, MA 02140, USA.
Michelle I LinVor Biopharma, Cambridge, MA 02140, USA.
Huanying Gary GeVor Biopharma, Cambridge, MA 02140, USA.
Amanda HalfondVor Biopharma, Cambridge, MA 02140, USA.
Shu WangVor Biopharma, Cambridge, MA 02140, USA.
Mark B JonesVor Biopharma, Cambridge, MA 02140, USA.
Julia EtchinVor Biopharma, Cambridge, MA 02140, USA.
Gabriella AngeliniVor Biopharma, Cambridge, MA 02140, USA.
Juliana Xavier-FerrucioVor Biopharma, Cambridge, MA 02140, USA.
Jessica LisleVor Biopharma, Cambridge, MA 02140, USA.
Kienan SalvadoreVor Biopharma, Cambridge, MA 02140, USA.
Yonina KeschnerVor Biopharma, Cambridge, MA 02140, USA.
Hannah MagerVor Biopharma, Cambridge, MA 02140, USA.
Julian SchererVor Biopharma, Cambridge, MA 02140, USA.
Jianxin HuVor Biopharma, Cambridge, MA 02140, USA.
Siddhartha MukherjeeDepartment of Medicine, Columbia University Irving Cancer Research Center, Columbia University, New York, NY 10032, USA.
Tirtha ChakrabortyVor Biopharma, Cambridge, MA 02140, USA.
Columbia University Irving Medical Center · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Immunotherapy of acute myeloid leukemia (AML) has been challenging because the lack of tumor-specific antigens results in "on-target, off-tumor" toxicity. To unlock the full potential of AML therapies, we used CRISPR-Cas9 to genetically ablate the myeloid protein CD33 from healthy donor hematopoietic stem and progenitor cells (HSPCs), creating tremtelectogene empogeditemcel (trem-cel). Trem-cel is a HSPC transplant product designed to provide a reconstituted hematopoietic compartment that is resistant to anti-CD33 drug cytotoxicity. Here, we describe preclinical studies and process development of clinical-scale manufacturing of trem-cel. Preclinical data showed proof-of-concept with loss of CD33 surface protein and no impact on myeloid cell differentiation or function. At clinical scale, trem-cel could be manufactured reproducibly, routinely achieving >70%

Indexed as

acute myeloid leukemiaCD33CRISPR-Cas9genome engineeringhematopoietic stem cellimmunotherapyIND-enabledpharmacology/toxicology

Identifiers

PMID38027064
PMCPMC10643325
OpenAlexW4387611140

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.