On Tuesday, April 6th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s January 2021 draft industry guidance on “Human Gene Therapy for Neurodegenerative Diseases.” In its comments, BIO stated that while the guidance covers many critical development issues for gene therapies, it is not specific to gene therapies for neurodegenerative diseases, and further requests that the Agency consider the potential impact of this guidance on gene therapy programs outside of neurodegenerative diseases. To this end, BIO encourages the issuance of (1) broad guidance on gene therapies that includes information that is applicable across diseases and (2) publication of focused and brief disease-specific guidance to provide information to Sponsors on disease-specific issues, as appropriate. Since aspects of this guidance may be relevant to cell therapies, BIO recommends that FDA develop a guidance or Q&A document that is quickly and easily updated, and consolidates cell and gene therapy policy with broad applicability into a single document. In the full comment letter, BIO also provides specific comments on the following topics: Gene Therapies and Accelerated Approval, Preclinical Studies, Chemistry Manufacturing and Controls (CMC), and Clinical Studies. 

Dear Secretary Buttigieg, National Climate Advisor McCarthy, and Director Deese: The undersigned organizations, representing aviation industry stakeholders including passenger and cargo carriers, business and general aviation, aircraft and engine manufacturers, airports, pilots, flight attendants, and aviation fuel suppliers as well as airline passengers and sustainable aviation fuel (SAF) producers within the advanced biofuels industry, write today to urge the Biden-Harris Administration to support the inclusion of a SAF-specific blender’s tax credit in the American Jobs Plan as you work with Congress to address infrastructure and the climate challenge. Congress has already indicated support for a SAF blender’s tax credit and recognized such a credit as the most efficient way to decarbonize the aviation industry while creating green jobs and opportunity for other sectors to join the clean energy economy.

On Friday, March 26th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s public meeting announcement titled, “Best Practices for Development and Application of Disease Progression Models.” BIO appreciates that FDA is seeking input from stakeholders on three key areas, including: the development and application of different types of disease progression models; modeling natural history of disease, specifically methodological considerations and challenges in characterizing the natural relationship between pharmacodynamic markers and clinical outcomes; and clinical trial simulations based on disease progression/natural history models to support drug development and regulatory decisions. Within each topic area, BIO proposed a list of specific issues that would highlight the opportunities and limitations in the development and application of disease progression models. In its comments, BIO also encouraged FDA to include case study examples from the public domain that would be illustrative of disease progression models, and to invite sponsors to present case examples that may not yet be in the public domain, including those that highlight both positive outcomes, lessons learned from negative outcome use cases, and use cases from the Model Informed Drug Development (MIDD) pilot program. To support this discussion, BIO members are willing to offer case study examples for inclusion in the agenda topics. BIO further encourages FDA to develop hypothetical case examples based on Agency experience that illustrate key issues and serve to generate discussion on how those challenges may be overcome.

The Biotechnology Innovation Organization is delighted to support the Inventor Diversity for Economic Advancement Act of 2021 and urge swift enactment of this important measure.