• How Are Cell And Gene Therapies Changing Drug Development Models?
    How Are Cell And Gene Therapies Changing Drug Development Models?

    A range of factors — including small patient populations, complex manufacturing processes, and lack of specialized expertise — are positioned to both drive up costs and require new options for stakeholder engagement and risk sharing along the development pathway. New approaches in development are needed to support the next generation of novel drugs on the horizon.

  • Regulators, Standards Groups Address Emerging Tech In Biopharma
    Regulators, Standards Groups Address Emerging Tech In Biopharma

    Manufacturers, suppliers, and regulators have recognized that introducing new technologies also introduces a degree of unfamiliarity and uncertainty.

  • What’s The Right Pricing Strategy For Cell And Gene Therapies?
    What’s The Right Pricing Strategy For Cell And Gene Therapies?

    This article highlights the diverse interests and range of opinions about pricing innovative and often high-cost therapies, but also demonstrates that most players in the sector see an urgent need for new pricing models to accommodate cell and gene therapy products.

Building New Business Models To Support Cell And Gene Therapy R&D
Building New Business Models To Support Cell And Gene Therapy R&D

Gene and cell therapy research has progressed despite the considerable challenges that require new approaches to the standard drug-development process and timeline. Here are some alternate development and financing models to consider.

  • New Microbiological Methods In Bioprocessing
    New Microbiological Methods In Bioprocessing

    Testing for evidence of microbial contamination in bioprocessing has a long history — and can be expensive, slow, and burdensome. So suppliers, testing facilities, and regulators have been seeking improvements.

  • Spark’s Luxturna Gene Therapy: What Can We Learn From Its Development & Commercialization?
    Spark’s Luxturna Gene Therapy: What Can We Learn From Its Development & Commercialization?

    Part 2 of this two-part article reviews lessons from Spark Therapeutics’ pivotal program for Luxturna, a gene therapy approved for the treatment of patients with retinal dystrophy associated with confirmed biallelic mutation in the RPE65 gene, and summarize key considerations for the clinical development and commercialization of gene therapies.

  • The Gene Therapy Pipeline — And The Biggest Challenges Facing Developers
    The Gene Therapy Pipeline — And The Biggest Challenges Facing Developers

    With several first-in-class gene therapies now approved, distinct new challenges are now coming into focus. This article provides a high-level overview of the gene therapy pipeline for non-oncology rare diseases and discuss key issues impacting clinical development.

  • Orphan Drug Commercial Models
    Orphan Drug Commercial Models

    This article posits that the orphan market is actually a collection of seven distinct business model archetypes that are driven by key disease, patient, and market characteristics. By disaggregating and characterizing these business models and their key success factors/risks, readers may be able to focus their corporate strategies to optimize the value and impact of their orphan therapies.

  • As Cancer Immune Therapies Advance, 4 Biomarker Trends To Watch
    As Cancer Immune Therapies Advance, 4 Biomarker Trends To Watch

    Despite progress, the current patient response rates and side effects associated with immunotherapies have created a sense of urgency to more accurately identify which patients would most benefit from a particular treatment option.

     

  • 4 Steps For Successful Tech Transfer Of Gene And Cell Therapy Products
    4 Steps For Successful Tech Transfer Of Gene And Cell Therapy Products

    As the gene and cell therapy sector develops and its products start to move from development to commercial manufacture — from smaller or academic establishments and development laboratories to larger facilities with greater capacity — the requirement for the technology transfer of these products will only increase.

  • Environmental Risks & The Life Science Supply Chain: Lessons Learned From Hurricane Maria
    Environmental Risks & The Life Science Supply Chain: Lessons Learned From Hurricane Maria

    Looking back at the hurricane season from the perspective of supply chain risk — and specifically risks that have impacted the life sciences supply chain — there are many lessons to be learned.

  • An Analysis Of FDA FY2017 Drug GMP Warning Letters
    An Analysis Of FDA FY2017 Drug GMP Warning Letters

    FY2017 saw another year of increase in the number of drug GMP warning letters issued by the FDA, though not as dramatic a difference as between FY2015 and FY2016. This article presents a detailed summary of those warning letters, as well as a comparison of trends since fiscal year 2013.

  • 3 Keys To Scale-Up CAR T-Cell Therapy Manufacturing
    3 Keys To Scale-Up CAR T-Cell Therapy Manufacturing

    Kymriah and Yescarta have overcome the first hurdle to commercialization by receiving the FDA’s stamp of approval. Now, Gilead and Novartis must prove they can handle the supply chain complexities involved with manufacturing and distributing these personalized, time-sensitive therapies to wider patient populations.

  • 5 Keys To Aseptic Processing Improvement & Efficiency
    5 Keys To Aseptic Processing Improvement & Efficiency

    In recent years, we’ve seen a rise in regulatory concern over adequately determining the risk factors that challenge aseptic and sterile products processing. There needs to be a renewed emphasis on assurance and controls, including leveraging the best available technologies and conducting thorough, science-based risk assessments of processes.

  • Commissioner Gottlieb, CAR T-Cells, & The Future Of Rare Disease Therapies
    Commissioner Gottlieb, CAR T-Cells, & The Future Of Rare Disease Therapies

    This article explores some of the policy changes happening now in the regulatory rare disease space as the FDA’s leadership team implements these changes at the agency.

  • Lessons In Navigating The Complex World Of Orphan Drug Commercialization
    Lessons In Navigating The Complex World Of Orphan Drug Commercialization

    The Orphan Drug Act of 1983 (ODA) has been wildly successful in its primary goal of spurring innovation that has led to lifesaving therapies. Unfortunately, the design of the ODA and several of the incentives the act provides have led to a host of unintended consequences.

  • Cancer Breakthroughs: A Look to The Past Can Be A Look To The Future
    Cancer Breakthroughs: A Look to The Past Can Be A Look To The Future

    Medical breakthroughs in the treatment of cancer have arrived at an accelerated pace. Seemingly each day, a new “next-generation” technology makes headline news, or a CEO touts a treatment as a game changer. While many of these will miss, others will succeed and undoubtedly make a difference in the lives of thousands of patients and their families.

CELL AND GENE INDUSTRY EVENTS

Business of Regenerative Medicine Conference July 17 - 18, 2018
Philadelphia, PA
The Bioprocessing Summit 2018 August 13 - 17, 2018
Boston, MA
2018 PDA Cell and Gene Therapy Conference October 23 - 24, 2018
Bethesda, MD
Stem Cell Research, Cell and Gene Therapy November 9 - 10, 2018
Atlanta, GA
Phacilitate Leaders World January 22 - 25, 2019
Miami, FL
Patients As Partners March 11 - 12, 2019
Philadelphia, PA
Car-T Congress USA March 20 - 22, 2019
Boston, MA
American Society of Gene & Cell Therapy (AGCST) April 29, 2019 - May 2, 2019
Washington, DC
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