• 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

    FDA Offers Guidance on Nicotine Replacement Therapies

    The US Food and Drug Administration (FDA) on Thursday issued draft guidance providing recommendations to drugmakers looking to develop nicotine replacement therapy (NRT) products to help patients quit smoking.   The 19-page draft guidance comes after FDA held a public consultation on its approach to evaluating NRT products in November 2017 and a public hearing on the matter in January 2018.   FDA says the draft guidance “takes into consideration the feedback re...
  • 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...
  • Regulatory NewsRegulatory News

    BIO Seeks Clarity on FDA’s Gene Therapy Draft Guidances

    Industry group BIO recently submitted comments on a series of new draft guidance documents on gene therapy released by the US Food and Drug Administration (FDA) in July. The six guidance documents focus on developing hemophilia, rare disease and retinal disorder gene therapies, and also include one on chemistry, manufacturing and control (CMC) information, one on long term follow-up observational studies collecting data on adverse events and one on the testing of retr...
  • Regulatory NewsRegulatory News

    FDA Approves New Gene Therapy Targeting Specific Mutation

    The US Food and Drug Administration (FDA) on Tuesday approved a new gene therapy from Spark Therapeutics for the treatment of children and adult patients with an inherited form of vision loss that may result in blindness. The approval comes two months and a week after an advisory committee of outside experts unanimously voted in favor of approving the treatment for the condition which affects about 6,000 worldwide. The price of the new gene therapy, which Leerink...
  • Feature ArticlesFeature Articles

    Resolving Gene Editing Technology's Ethical and Regulatory Challenges

    This article provides an overview of biomedical applications of gene editing technology, addresses ethical and regulatory challenges associated with its implementation for therapeutic development, and proposes approaches for overcoming these challenges. Introduction Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology is a genetic "editing" tool aimed at altering DNA sequences and modifying gene function. It offers promising opportunities for ...
  • Regulatory NewsRegulatory News

    Experts Tell Senate Committee Current Gene Editing Regulations are Appropriate

    At a Senate Health, Education, Labor and Pensions (HELP) hearing in Washington, D.C. on Tuesday, experts explained that the current regulatory framework for gene editing in the US is appropriate and cautioned that an overly strict approach could drive research to other countries. Specifically, the hearing focused on CRISPR/Cas9, which is a gene editing tool that scientists can use to quickly and accurately edit DNA in a variety of applications. While there are curr...
  • Regulatory NewsRegulatory News

    EMA, European Commission Look to Foster Development of Advanced Therapies

    The European Medicines Agency (EMA) and European Commission on Friday published a joint action plan to further the development of new cell and gene therapies, as well as other advanced therapy medicinal products (ATMPs). The plan, which features the release of several new and revised guidelines comes as on the international level, a regular forum for dialogue has been established to share experiences on advanced therapies between EMA, the US Food and Drug Administr...
  • Regulatory NewsRegulatory News

    FDA Panel Votes Unanimously in Favor of Gene Therapy to Treat Rare Eye Disorder

    Philadelphia-based Spark Therapeutics took its gene therapy before the US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee on Thursday and the panel voted 16 to 0 in favor of approving the subretinal injection, representing a major step forward for adeno-associated virus gene therapies. The therapy, known as Luxturna (voretigene neparvovec), has been granted priority review by FDA (the agency has until 12 January 2018 t...
  • Regulatory NewsRegulatory News

    'Historic Action': FDA Approves First CAR-T Therapy

    The US Food and Drug Administration (FDA) on Wednesday approved the first gene therapy, Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Dubbed an "historic action" by the agency, the approval comes a little more than a month after FDA's Oncologic Drugs Advisory Committee voted unanimously , 10 to 0, in favor of the benefit-risk profile for the first of this new kind of cancer th...
  • Regulatory NewsRegulatory News

    FDA to Unveil New Regulatory Framework for Stem Cell Therapies

    The US Food and Drug Administration's (FDA) Commissioner Scott Gottlieb said Monday the agency will advance a new framework to better regulate stem cell therapies this fall. "This comprehensive policy will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency's current authority, and then define an efficient process for how these products should be evaluated for safety and effectiveness," Gottli...
  • Feature ArticlesFeature Articles

    Is Increasing Antibiotic Resistance Driving Bacteriophage Therapy Closer to Being a Reality?

    This article reviews the history of bacteriophage discovery, biology, and potential medical uses, and discusses therapeutic pros and cons compared to and potentially replacing antibiotics due to increasing antibiotic resistance. The article also highlights the future of phage therapy and the need for more scientific investigation and clinical trials prior to approval for human use and the possible patenting and regulatory issues impacting bacteriophage development. The U...