Evidence map›Paper›PMID 41346304›Full record

ReviewClinical and translational science2025

Cell and Gene Therapy: Transforming Treatment Paradigms for Patient-Centric Care.

Sojeong Yi, Chia-Yung Wu, Avery McIntosh, Maria E Santaella, Tara M Robinson, Michael Z Liao

Abstract readReview
In one paragraph

Review in Clinical and translational science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Sojeong YiAlnylam Pharmaceuticals, Inc, Cambridge, Massachusetts, USA.ORCID 0000-0001-6659-1585
Chia-Yung WuLima Hui BioConsulting, Seattle, Washington, USA.
Avery McIntoshPfizer, Inc, Collegeville, Pennsylvania, USA.ORCID 0000-0002-4164-502X
Maria E SantaellaNational Bleeding Disorders Foundation, New York, New York, USA.ORCID 0000-0003-2029-677X
Tara M RobinsonTakeda Development Center Americas, Inc, Cambridge, Massachusetts, USA.
Michael Z LiaoThird Arc Bio, Inc, Lower Gwynedd, Pennsylvania, USA.ORCID 0000-0003-0684-0150

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Cell and gene therapies (CGTs) are transforming medicine by offering potential cures for diseases previously considered untreatable. Despite rapid advancements, challenges remain in optimizing efficacy and safety and ensuring patient accessibility and preference due to high costs and clinical uncertainties, particularly for rare diseases and one-time administration. The American Society of Clinical Pharmacology & Therapeutics (ASCPT) held a CGT satellite conference in 2025, titled "Cell and Gene Therapy: Transforming Treatment Paradigms for Patient-Centric Care." This manuscript summarizes the conference, covering gene therapies and T-cell immunotherapies from scientific, clinical, and patient-centered perspectives. Key topics on gene therapy included "platformization" to streamline development, lessons from adeno-associated virus-based gene therapies for hemophilia from patient and clinical perspectives, clinical pharmacology, and model-informed drug development (MIDD) considerations. The conference also highlighted T-cell immunotherapies including chimeric antigen receptor T therapy (CAR T), focusing on factors affecting cellular kinetics, efficacy, and safety, as well as emerging allogeneic CAR T for autoimmune diseases and MIDD strategies to optimize therapy design and clinical outcomes.

Indexed as

Cell- and Tissue-Based TherapyGenetic TherapyPatient-Centered CareDependovirusHemophilia AHumansImmunotherapy, AdoptiveT-Lymphocytescell‐ and tissue‐based therapygene editinggenetic therapyimmunotherapymodel informed drug development

Identifiers

PMID41346304
PMCPMC12678861

What OpenQuestion holds

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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.