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  • Regulatory NewsRegulatory News

    Cell and gene therapies: FDA official on COVID-19 impact

    Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US Food and Drug Administration (FDA) official discussed the impact of the coronavirus disease (COVID-19) pandemic on the agency’s work related to cell and gene therapies.   “Before the pandemic, we were seeing a huge escalation in the workload that we have to make adjustments for,” said Wilson Bryan, director of the Office of Tissues and Advanced Therapies at the Center for Biologics Eval...
  • Feature ArticlesFeature Articles

    Bespoke therapies – opportunities, challenges, and hope

    This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new...
  • Regulatory NewsRegulatory News

    Refine gene therapy follow-up guidance, expert says

    It’s time for regulatory agencies, academics and pharmaceutical companies to convene to refine guidance for long-term follow-up of patients receiving gene therapy. That was the message Anne-Virginie Eggimann, senior vice president for regulatory science at bluebird bio, Inc., brought to a policy session at the annual meeting of the American Society of Gene & Cell Therapy .   To date, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) hav...
  • Regulatory NewsRegulatory News

    Novartis Admits to ‘Mistake’ After Partial Clinical Hold Placed on Zolgensma Trial

    Novartis on Wednesday said that the US Food and Drug Administration (FDA) placed a partial hold on intrathecal clinical trials of its gene therapy Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) patients based on findings in a small preclinical animal study.   On Thursday, a Novartis spokesperson told Focus that a draft report of the preclinical safety findings was presented to the AveXis safety management team last March and the company “...
  • RoundupsRoundups

    Asia Regulatory Roundup: Hong Kong Proposes Regulatory Framework for Cell and Gene Therapies

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia.   Hong Kong Proposes Regulatory Framework for Cell and Gene Therapies   Hong Kong has proposed legislation to create a regulatory framework for advanced therapy products (ATPs), such as interventions based on cells, genes and tissues. The plan is to amend existing laws to create licensing, labeling and record-keeping requirements for ATPs.   Work to update the law ...
  • Regulatory NewsRegulatory News

    As Stem Cell Clinics Proliferate, E&C Leaders Seek to Hear FDA’s Plan

    A bipartisan group of representatives on the Energy & Commerce (E&C) committee sent a letter to US Food and Drug Administration (FDA) Acting Commissioner Ned Sharpless on Thursday seeking more details on the agency’s plan to slow the proliferation of stem cell clinics. While noting that FDA has agreed to exercise enforcement discretion for 36 months in a recent guidance, the letter also explains how CBER Director Peter Marks said in April that industry has shown “mo...
  • Regulatory NewsRegulatory News

    FDA Questions Arizona Stem Cell Company

    As part of efforts to crack down on companies selling unapproved stem cell products to treat a variety of diseases and conditions, the US Food and Drug Administration (FDA) on Thursday sent an untitled letter to Arizona-based R3 Stem Cell and its more than 50 affiliate centers or clinics. FDA raises concerns with R3 because its website makes unsubstantiated claims about the stem cell products, which FDA says appear to be human cell, tissue, or cellular or tissue-based p...
  • Regulatory NewsRegulatory News

    Drug Regulators Look to Harmonize How They Tackle Innovation

    The International Coalition of Medicines Regulatory Authorities (ICMRA) recently released a report on how various regulators worldwide are working together to better identify and address future regulatory challenges posed by new categories of therapeutics, like cell and gene therapies, and new tools for drug development, such as artificial intelligence (AI). The report, which is part of a wider effort to reduce duplicative work and increase harmonization among drug regu...
  • Regulatory NewsRegulatory News

    FDA to Step Up Stem Cell Enforcement, Look Into Pathway for Low-Risk Treatments

    US Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation Research (CBER) Director Peter Marks on Wednesday warned that the agency will step up its enforcement efforts against companies illegally marketing stem cell therapies.   Gottlieb and Marks also said the agency will look into new ways to “delineate an efficient development path” for low-risk stem cell therapies being developed by firms that have filed investigational n...
  • Regulatory NewsRegulatory News

    HMA Offers Recommendations on Complex Clinical Trials

    A subgroup of the EU’s Heads of Medicines Agencies (HMA) issued new recommendations Friday on conducting complex clinical trials.   The HMA’s Clinical Trials Facilitation Group (CTFG)—coordinated by the Danish Medicines Agency—identified eight key recommendations to establish a framework on the initiation and conduct of complex trials for the development of personalized medicine. They were prepared “in recognition of the fact that the development of personalized medici...
  • Regulatory NewsRegulatory News

    Asia Regulatory Roundup: China Floats Tighter Clinical Trial Oversight Following Gene Editing Scandal

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia.   China Floats Tighter Clinical Trial Oversight Following Gene Editing Scandal   China is planning to introduce a risk-based regulatory system for cell and gene therapies in the wake of the germline modification scandal. The system would require developers of high-risk therapies to get state-level clearance for clinical trials, while leaving local authorities to sign o...
  • Regulatory NewsRegulatory News

    CBER Guidance on Gene Therapies: What to Expect in 2019

    The US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) on Wednesday released plans for what guidance is to be expected in 2019, including six final guidance documents related to gene therapies. The gene therapy guidance documents ( draft versions were published in July ) the agency expects to finalize include: Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry Chemistry, Manufacturing, an...