Evidence map›Paper›PMID 40616579›Full record

ReviewCytotherapy2025

Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher.

Fyodor Urnov, Sadik Kassim, Kiran Musunuru, David Liu, Ann Lee, Luis Barrera, Pam Stetkiewicz, Julianne Bruno, Matthew Hewitt, Troy Lister and 9 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Cytotherapy, 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. Review
  2. Article
  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

19 authors.

Fyodor UrnovInnovative Genomics Institute, University of California, Berkeley, California, USA.
Sadik KassimDanaher Corporation, Washington, District of Columbia, USA.
Kiran MusunuruPerelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
David LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, Massachusetts, USA; Department of Chemistry and Chemical Biology; Harvard University, Cambridge, Massachusetts, USA; Howard Hughes Medical Institute, Harvard University, Cambridge, Massachusetts, USA.
Ann LeePrime Medicine, Cambridge, Massachusetts, USA.
Luis BarreraBeam Therapeutics, Cambridge, Massachusetts, USA.
Pam StetkiewiczArbor Biotechnologies, Cambridge, Massachusetts, USA.
Julianne BrunoCRISPR Therapeutics, Boston, Massachusetts, USA.
Matthew HewittCharles River Laboratories, Wilmington, Massachusetts, USA.
Troy ListerVerve Therapeutics, Boston, Massachusetts, USA.
Harry MalechNational Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Lindsay GaschCapstone Medical Writing & Communications, Lander, Wyoming, USA.
Matt DiverGalen/Atlantica, Boston, Massachusetts, USA.
Nicholas GertlerGalen/Atlantica, Boston, Massachusetts, USA.
Felix GrignonInternational Society for Cell & Gene Therapy, Vancouver, British Columbia, Canada.
Audrey LeInternational Society for Cell & Gene Therapy, Vancouver, British Columbia, Canada.
Michael LehmickeAlliance for Regenerative Medicine, Washington, District of Columbia, USA.
Vanessa Almendro-NavarroDanaher Corporation, Washington, District of Columbia, USA.
Josephine LembongAlliance for Regenerative Medicine, Washington, District of Columbia, USA. Electronic address: jlembong@alliancerm.org.

Funding

Intramural NIH HHS Z99 AI999999
6 · The paper itself

Abstract

Rare-disease therapeutics face viability challenges due to small patient populations and drug-development and regulatory frameworks that were not developed to address rapidly progressive or quickly fatal conditions. Because the majority of rare diseases are genetic in nature, gene-editing modalities offer substantial promise. This Scientific Exchange, co-hosted by the Alliance for Regenerative Medicine, the International Society for Cell and Gene Therapy, and Danaher Corporation in November 2024, set out to address the challenge of realizing the full promise of gene editing for rare-disease therapies by advancing platforms that leverage stable and reusable processes or components to develop multiple therapies. Through multi-stakeholder engagement and discussions of case studies in CRISPR/Cas nuclease, base, and prime editing, 4 key opportunities emerged that deliver value by holding platform elements constant and/or streamlining development steps: (1) consistent delivery vehicle; (2) consistent manufacturing; (3) benefit-risk appropriate quality requirements; and (4) expansive clinical trial designs. Together, these opportunities could yield up to 5-fold efficiency gains and result in substantial value creation for patients, regulators, and developers, potentially decreasing the time required to dose patients with a new gene-editing therapy from years down to 6 months.

Indexed as

Gene EditingGenetic TherapyRare DiseasesCRISPR-Cas SystemsHumansRegenerative Medicinebase editingCRISPRgene editingguide RNAplatformprime editingrare disease

Identifiers

PMID40616579
PMCPMC12641462

What OpenQuestion holds

Textmetadata
LicenceTDM
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.