Evidence map›Paper›PMID 40779197›Full record

ReviewTherapeutic innovation & regulatory science2025

Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases-by DahShu Innovative Design Scientific Working Group (IDSWG).

Chenkun Wang, Junrui Di, Mercedeh Ghadessi, Rui Tang, Caroline Mulatya, Daoyuan Shi, Tu Xu, Wenquan Wang, Chaoqun Mei, Susan Wang and 3 more

Abstract readReview
PubMed Publisher
In one paragraph

Review in Therapeutic innovation & regulatory science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

13 authors.

Chenkun WangVertex Pharmaceuticals, Inc., 50 Northern Avenue, Boston, MA, 02210, USA. Chenkun_Wang@vrtx.com.
Junrui DiDexcom Inc., San Diego, CA, 02139, USA.
Mercedeh GhadessiBayer U.S. LLC, Pharmaceuticals, 100 Bayer Boulevard, Whippany, NJ, 07981, USA.
Rui TangAstellas Pharma Inc, 441 Morgan Ave, Cambridge, MA, 02139, USA.
Caroline MulatyaREGENXBIO Inc., 9804 Medical Center Dr, Rockville, MD, 20850, USA.
Daoyuan ShiBlueRock Therapeutics, 238 Main St, Cambridge, MA, 02142, USA.
Tu XuNovo Nordisk, 75 Hayden Ave, Lexington, MA, 02421, USA.
Wenquan WangPfizer Inc., Collegeville, PA, 19426, USA.
Chaoqun MeiBristol Myers Squibb, Madison, NJ, 07940, USA.
Susan WangBoehringer Ingelheim Pharmaceuticals Inc., 900 Ridgebury Road, Ridgefield, CT, 06877, USA.
Bryan McCombPfizer Inc., 66 Hudson Blvd East, New York, NY, 10001, USA.
Robert A BeckmanLombardi Comprehensive Cancer Center and Innovation Center for Biomedical Informatics, Georgetown University Medical Center, Washington, DC, 20007, USA.
Gianna McMillanBioethics Institute at Loyola Marymount University, 1 LMU Drive, Los Angeles, CA, 90045, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The rapid advancement of cell and gene therapies (CGT) in the past ten years has inspired biopharmaceutical companies, biotechnologies, and nonprofits to tackle diseases that have traditionally been challenging to treat. Rare diseases, where roughly 80% have a genetic basis, have enjoyed this scrutiny, but the complexity of CGT trial design and implementation have proven challenging. This manuscript offers general guidance for CGT clinical development, current regulatory requirements and guidelines governed by FDA and EMA, considerations around preclinical development, safety monitoring and the need for long-term monitoring and follow up.

Indexed as

Cell- and Tissue-Based TherapyGenetic TherapyRare DiseasesClinical Trials as TopicHumansUnited StatesUnited States Food and Drug AdministrationCell and gene therapyClinical developmentRegulatory requirements and guidelinesSafety monitoring

Identifiers

PMID40779197

What OpenQuestion holds

Textmetadata
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.