Search Results for Gene therapy

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FDA Panel Votes Unanimously in Favor of First CAR-T Cancer Therapy By Zachary Brennan - Published 12 July 2017

The US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee on Wednesday voted unanimously, 10 to 0, in favor of the benefit-risk profile for the first of a new kind of cancer therapy, known as a Chimeric Antigen Receptor T-cell (CAR-T) therapy.

Categories: News, US, FDA, Government affairs, Human cell and tissue, Manufacturing, Quality, Regulatory intelligence, Regulatory strategy

Tags: CAR-T, Novartis, CTL019, gene therapy, cell therapy, ODAC

CBER Director Focuses on Flexibility to Advance Regenerative Medicines By Michael Mezher - Published 08 May 2017

Peter Marks, director of the US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research (CBER) on Thursday said his office is equipped to support the development of cutting edge treatments, such as cell and gene therapies.

Categories: News, US, CBER, Biologics and biotechnology, Human cell and tissue, Regulatory strategy

Tags: Regenerative Medicine, Cell and Gene Therapy, Advanced Therapies, RMAT Designation

Regulation and Oversight of Gene Therapy in the US By Gregory Ramina - Published 27 February 2017

This article discusses the evolution, potential uses and regulatory aspects of gene therapy and includes funding, delivery mechanisms, bioethics and steps toward clinical trial implementation.

Categories: Features, US, FDA, Biologics and biotechnology, Human cell and tissue

Tags: Gene therapy,

FDA Withdraws Proposed Rule on Public Disclosure of Info on Unapproved Gene Therapies By Zachary Brennan - Published 10 November 2016

The US Food and Drug Administration (FDA) on Thursday announced it is withdrawing a proposed rule from 2001 that would have required the public disclosure of summaries of safety and effectiveness data, in addition to other information, from pre-market clinical trials of gene therapies and transplanted non-human tissues to humans, known as xenotransplantation.

Categories: News, US, CBER, Biologics and biotechnology, Clinical, Crisis management, Due Diligence, Government affairs, Human cell and tissue, Preclinical, Research and development

Tags: gene therapy, xenotransplantation, BIO, Public Citizen

EMA to Continue Adaptive Pathways Program as Pilot Ends By Zachary Brennan - Published 03 August 2016

The European Medicines Agency (EMA) on Wednesday published a final report on its experience from its adaptive pathways pilot project, noting that a few aspects of the program need to be further improved and refined moving forward.

Categories: News, Europe, EMA, Biologics and biotechnology, Drugs, Government affairs, Regulatory intelligence, Regulatory strategy

Tags: adaptive pathways, EMA pilot project, Blue, gene therapy, real world data

European Commission to Overhaul Concept of ‘Similar’ Medicines Within Context of Orphan Legislation By Zachary Brennan - Published 29 July 2016

The European Commission on Friday proposed to review the concept of “similar medicinal products” in the context of its orphan legislation as part of a wider effort to adapt the text to technical progress.

Categories: News, Europe, EC, Active pharmaceutical ingredients, Biologics and biotechnology, Drugs, Government affairs, Orphan products

Tags: orphan drug legislation, European Commission, gene therapy, ATMP

EC Report Analyzes Regulatory Frameworks for Advanced Therapies in US, Canada, Japan and Korea By Zachary Brennan - Published 06 July 2016

The European Commission report released Wednesday dissects the similarities in how gene-, cell- and tissue-based advanced therapies are regulated across the four different geographic regions, though ongoing research projects in such therapies are heavily concentrated in the US and Japan.

Categories: News, Asia, Canada, Europe, US, EC, EMA, FDA, Health Canada, MHLW, PMDA, Biologics and biotechnology, Clinical, Government affairs, Human cell and tissue

Tags: advanced therapies, gene therapies, reports on advanced therapies, gene therapy regulation

Advanced Therapies: Stakeholders Call for EMA Changes on Regulations, Manufacturing By Zachary Brennan - Published 03 June 2016

The European Medicines Agency (EMA) on Friday released a report outlining the concerns and regulatory changes described by developers, manufacturers and investors in advanced therapy medicinal products (ATMPs), which include gene therapies, tissue-engineered products and somatic cell therapies, at a meeting last week.

Categories: News, Europe, EC, Biologics and biotechnology, Blood, Clinical, Combination products, Crisis management, Government affairs, Human cell and tissue, Manufacturing, Quality, Regulatory intelligence, Regulatory strategy, Research and development

Tags: ATMPs, gene therapy manufacturing, cell therapy, advanced therapies

Senate Bill Seeks to Create Standards Body for Regenerative Medicine By Michael Mezher - Published 15 January 2016

Sen. Tammy Baldwin (D-WI) introduced a bill on Tuesday to promote the development of regulatory science and standards for regenerative medicine and advanced therapies.

Categories: News, US, CBER, Biologics and biotechnology, Government affairs, Human cell and tissue

Tags: Gene therapy, Cell therapy, Regenerative medicine, Advances therapies, Senator Tammy Baldwin, Michael Werner

Cancer Gene Therapies: FDA Officials Highlight Regulatory Approaches By Zachary Brennan - Published 25 November 2015

As gene therapies begin to get a foothold among other cancer treatments, the US Food and Drug Administration (FDA) is taking a more flexible, data-driven approach for preclinical testing programs for these biologically complex products, according to a new review from five FDA officials in Cancer Gene Therapy.

Categories: News, US, FDA, Biologics and biotechnology, Blood, Business Skills, Due Diligence, Human cell and tissue, Preclinical, Quality, Regulatory strategy

Tags: cancer gene therapies, CBER, FDA regulations on gene therapy, regulatory issues for CBER

EMA Issues New Guideline on Gene Therapy By Michael Mezher - Published 21 May 2015

The European Medicines Agency (EMA) is seeking feedback on a new draft guideline aimed at clarifying the scientific evidence necessary to support the authorization of new gene therapies.

Categories: News, Europe, EMA, ICH, Biologics and biotechnology, Clinical, Human cell and tissue, Preclinical, Quality, Regulatory strategy

Tags: Gene therapy, Advanced therapy medicinal product, ATMP, Glybera

Following Negative Comments by Reviewer, EU Committee OK's Gene Therapy By Michael Mezher - Published 06 May 2015

Last month, German regulators postponed their review of UniQure's gene therapy Glybera after the European Medicines Agency (EMA) rapporteur assigned to the product claimed new data showed Glybera lacked efficacy and called for it to be reassessed.

Categories: Clinical, Postmarket surveillance, Regulatory strategy, Submission and registration

Tags: Committee for Advanced Therapies, CAT, G-BA, Federal Joint Committee, Glybera, UniQure, Gene therapy

First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New Report By Michael Mezher - Published 17 April 2015

Regulators at the German Federal Joint Commission (G-BA) have postponed their review of the gene therapy Glybera, Reuters reports. The decision was made after the European Medicines Agency (EMA) rapporteur assigned to Glybera found the product lacked efficacy and called for it to be reassessed.

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Orphan products, Postmarket surveillance, Submission and registration

Tags: Glybera, Gene therapy, Committee on Advanced Therapies, CAT, Federal Joint Commission, G-BA

FDA Wants Sponsors of Biotech Products to Assess Product's Impact on Environment By Alexander Gaffney, RAC - Published 23 June 2014

The US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research (CBER) has released a new draft guidance document meant to clarify when companies need to conduct an Environmental Assessment (EA) for gene therapy and other recombinant products.

Categories: News, US, CBER, Biologics and biotechnology, Submission and registration

Tags: Environmental Assessment, Guidance, Draft Guidance, Refuse to File, GTVVs, GTVV, Recombinant, Gene Therapy, Vector Vaccine, Biotechnology

Cell and Gene Therapy Guidance Finalized by FDA, Calls for Early and Frequent Communication By Alexander Gaffney, RF News Editor - Published 25 November 2013

Categories: US, FDA

Tags: Cell Therapy, Gene Therapy, guidance

FDA Extends Comment Period on Cell and Gene Therapy Guidance by Six Months By Alexander Gaffney, RF News Editor - Published 19 November 2013

Categories: US, FDA

Tags: Cell Therapy, Gene Therapy, AdComm, draft guidance

Safety Main Challenge to Preparing INDs for Cellular and Gene Therapy Products, Says FDA By Alexander Gaffney, RF News Editor - Published 03 July 2013

Categories: US, FDA

Tags: Gene Therapy, IND, draft guidance

Draft Guidance on Cell, Gene Therapy Products Establishes General Framework By Alexander Gaffney, RF News Editor - Published 29 November 2012

Categories: FDA, Preclinical

Tags: Cell Therapy, CTG, Gene Therapy, BLA, IND, Latest News, draft guidance

CHMP Recommends EU's First Gene Therapy Product By Alexander Gaffney - Published 20 July 2012

Categories: EMA

Tags: Glybera, Gene Therapy, European Commission, Latest News, CHMP