New public-private consortium will tackle gene therapies for rare diseases

Regulatory NewsRegulatory News | 28 October 2021 |  By 

Two federal agencies are partnering with pharmaceutical companies and non-profit organizations to form a consortium aimed at boosting the development of gene therapies for rare diseases.
In a Wednesday announcement, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH) revealed the launch of the Bespoke Gene Therapy Consortium (BGTC). The consortium’s work will be managed by the Foundation for the NIH and sit within the NIH Accelerating Medicines Partnership program, which brings public and private resources to bear on a variety of disease conditions.
“By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them most,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research. “FDA is committed to developing a regulatory paradigm that can advance gene therapies to meet the needs of patients with rare diseases.”
One key goal of BGTC will be to take a close look at adeno-associated virus (AAV) vectors, breaking down and understanding the biology and the mechanistic steps that go into AAV vector production, delivery, and subsequent intracellular activation of the genes delivered by AAV vectors.
From this learning, researchers in the BGTC consortium will eventually be able to use standardized analytic testing methods that can be applied for a variety of manufacturing methods for viral vectors, increasing process efficiency.
In another BGTC initiative, the consortium will select from four to six monogenetic rare diseases that currently have no gene therapies or other treatments in commercial development. BGTC will initiate clinical trials for the rare conditions that are selected, which the announcement says should already have “substantial groundwork in place to rapidly initiate preclinical and clinical studies.”
BGTC also plans to examine the current regulatory scheme for gene therapies and seek efficiencies that can speed development by streamlining the FDA approval process. Standardization of toxicology testing for gene therapies at the preclinical stage is one example of the efficiencies that the consortium will seek to achieve.
FDA’s ‘N of 1’ draft guidance
Bespoke gene therapies received the attention of FDA earlier this year, when the agency issued a draft guidance document that gives developers of anti-sense oligonucleotides for so-called “N of 1” therapies a high-level view of FDA’s thinking on the rapidly developing field. The scope of that draft guidance is limited to the pre-investigational new drug (IND) and IND submission stages. (RELATED: 'N of 1' therapies addressed in draft FDA guidance, Regulatory Focus 05 January 2021)
In a comment on the draft guidance, the American Society for Cell and Gene Therapy pointed out that sponsors of bespoke gene therapies for very small populations may have little experience with the regulatory process, FDA’s provision of detailed procedural information in the draft guidance is useful.
The National Organization for Rare Disorders (NORD) also left a comment on the draft guidance that highlighted the need to involve patients and caregivers in rare disease therapy research and development, because they are often uniquely positioned to understand key aspects of disease status and progression.
The Association for Regenerative Medicine (ARM), in its comments on the draft guidance, asked for more specificity from FDA on the agency’s expectations for later stages in the gene therapy development process, including “regulatory considerations to support first in human exposure/treatment, collection of data on efficacy, short and long term follow up, treatment outcomes, and small-scale manufacturing that could inform future patient treatments and, eventually, clinical trials to support an FDA approved product for these patients.”
BGTC funding, leadership, partners
The joint NIH-FDA announcement of the BGTC launch points out that although each rare disease may affect a very small number of people – sometimes just a handful – there are many rare diseases that, taken together, affect many people.
“Rare diseases affect 25 to 30 million Americans, but because any given rare disorder affects so few patients, companies often are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring individualized gene therapy treatments for a single disease to market,” said Joni L. Rutter, acting director of NIH’s National Center for Advancing Translational Sciences (NCATS), in the announcement. NCATS, the lead NIH institute in moving the initiative forward, has also developed the Platform Vector Gene Therapy (PaVe-GT) pilot project, also aimed at streamlining gene therapy development and clinical delivery.

“The BGTC aims to make it easier, faster and less expensive to pursue bespoke gene therapies in order to incentivize more companies to invest in this space and bring treatments to patients,” said Rutter.
The partnership will receive about $76 million over 5 years, with nearly $40 million of that figure coming from NIH.
Private partners in the BGTC initiative include:
  • Biogen
  • Janssen Research & Development
  • Novartis Institutes ofr BioMedical Research
  • Pfizer
  • Spark Therapeutics
  • Takeda
  • Taysha Gene Therapies
  • Thermo Fisher Scientific
  • Ultragenic Pharmaceutical
Non-profit organizations participating in the consortium include:
  • Alliance for Regenerative Medicine
  • American Society of Gene and Cell Therapy
  • CureDuchenne
  • National Organization for Rare Disorders
  • National institute for Innovation in Manufacturing Biopharmaceuticals
FDA announcement


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