Gene Therapy Developers Issue Principles for Human Genome Editing

The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force released a set of principles for human genome editing this month.

By Michael Mezher | Aug 9, 2019 | Regulatory Focus

The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the development of gene therapies or gene-editing technology.
 
While the principles endorse somatic cell gene editing and the development of regulatory standards for gene editing, the document asserts that it is too early to support any form of human germline gene editing due to unanswered ethical, legal and safety questions.
 
“As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles,” said ARM CEO Janet Lambert.
 
Background
 
The framework comes less than a year after Chinese scientist He Jiankui announced he had edited the genes of infant twin girls using CRISPR in an attempt to make them more resistant to HIV infection. Jiankui’s announcement prompted swift international backlash, with regulators in China proposing rules to restrict gene editing in humans and experts around the world calling for a moratorium on human germline editing.
 
The World Health Organization (WHO) also reacted to the announcement by forming an advisory panel on human genome editing, which called for the development of a central registry for all human genome editing studies last March.
 
A Framework for Gene Editing
 
Among the five principles included in the framework are an endorsement of therapeutic applications of somatic cell gene editing; support for the development of gene editing standards and “evolving” regulatory frameworks for gene editing techniques; and opposition to human germline gene editing until unresolved scientific, legal and ethical questions are addressed at an international level.
 
The companies endorsing the principles include Audentes, bluebird bio, BlueRock, Caribou Biosciences, Casebia Therapeutics, CRISPR Technologies, Editas, Homology Medicines, Intellia Therapeutics, LogicBio, Precision Biosciences, Sangamo and Tmunity.
 
In addition to opposing human germline gene editing, the task force also opposes the implantation of human embryos that carry gene modified cells.
 
“Regulated, clinical validation of somatic cell-based gene editing technologies for non-inherited genetic modification is, and should remain, the primary objective of the therapeutic development community and, in contrast to germline gene editing, offers the most acceptable near-term path to potentially transformative therapeutic benefits for patients,” the task force writes.
 
The principles offer support for efforts by the National Institute of Standards and Technology (NIST) Genome Editing Consortium, US Pharmacopoeia, International Organization for Standardization (ISO) and other standards bodies that are working to develop gene editing standards that could support the development and assessment of gene therapies.
 
The task force also argues that existing regulatory frameworks from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) offer appropriate oversight for somatic cell gene therapies, but caution that “arbitrary and ancillary oversight bodies or processes” could delay the development of promising new treatments.

TRNDS 2019

University of Rochester
Saunders Research Building
Helen Wood Hall Auditorium
255 Crittenden Blvd
Rochester, NY 14642

Date: Friday, September 13
Time: 7:30 am – 4:00pm

TRNDS News

Sign-up for TRNDS updates and rare disease news

  • New Grants will Accelerate Clinical Trials in Rare Neurological Disorders
    READ MORE
  • Neurogene Offers Access to Genetic Testing for Lysosomal Storage Disease
    READ MORE
  • NIH launches 5-year, $10 million study on acute flaccid myelitis
    READ MORE
  • Study refines ALS risk among first-degree relatives of patients with disease
    READ MORE
  • NCATS Director Warns of 'Poorly Understood Public Health Implications of Rare Diseases'
    READ MORE
  • Speeding Up Drug Discovery for Brain Diseases
    READ MORE
  • NIH-Funded Project Aims to Build a ‘Google’ for Biomedical Data
    READ MORE
  • Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
    READ MORE
  • Jsyne Gershkowitz, Amicus Therapeutics, TRNDS 2019
    (Podcast) Get to know Jayne Gershkowitz, Chief Patient Advocate at Amicus Therapeutics
    READ MORE
  • PJ Brooks, NCATS, NIH, TRNDS
    Meet TRNDS 2019 Speaker PJ Brooks
    READ MORE
  • Rare Pediatric Disease PRVs: FDA Updates Guidance
    READ MORE
  • Batten Disease Study
    Neurogene Opens Natural History Study of CLN7, CLN5 Diseases at UT Southwestern
    READ MORE
  • C-Path and NORD Collaborate to Launch Rare Disease Data Platform
    READ MORE
  • TRNDS 2019 speaker Tauna Batiste, BDSRA
    Meet TRNDS Speaker Tauna Batiste
    READ MORE
  • HD Patients Prefer Less Invasive, No-Placebo Gene Therapy Trials
    READ MORE
  • Evidation & Eli Lilly Study Uses Apple Devices, Apps to Predict Cognitive Impairment
    READ MORE
  • 'Dr. Google' Helps Some Patients Diagnose a Rare Disease
    READ MORE
  • Antisense Drugs for HD, ALS & Prion Could Meet the Need for Brain Treatments
    READ MORE
  • Ultragenyx Partners with GeneTx to Advance Treatment for Angelman Syndrome
    READ MORE
  • Netflix's Medical Investigation Docuseries 'Diagnosis'
    READ MORE
  • NIH All of Us Project Tops 270,000 Sign-ups
    READ MORE
  • FDA Cancer Office Taps Syapse for Real-world Evidence Development
    READ MORE
  • Gene Linked to Rare Neurological Disorder May Play a Role in Alzheimer’s
    READ MORE
  • Gene Therapy Developers Issue Principles for Human Genome Editing
    Gene Therapy Developers Issue Principles for Human Genome Editing
    READ MORE
  • Forget Single Genes: CRISPR Now Cuts & Splices Whole Chromosomes
    READ MORE
  • Benjjamin Schlatka MC10 TRNDS
    TRNDS Speaker Benjamin Schlatka, MC10
    READ MORE
  • TRNDS Danielle Edwards Jett Foundation
    TRNDS Speaker Danielle Edwards, Jett Foundation
    READ MORE
  • His daughter has a rare disorder. He’s developing a novel gene therapy.
    READ MORE
  • A Doctor Tested a New Treatment on Himself, Now it's Saving Other Lives
    READ MORE
  • Study Presents Clinical, Genetic Landscape of Pompe Disease
    READ MORE

Latest Tweets

TRNDS Speakers

Room Block

Hilton Garden Inn
Rochester/University
Medical Center

30 Celebration Drive
Rochester, NY 14620

Phone: 585-424-4404

TRNDS Rate: $149/night