Cell and Gene Therapies

Biologics and cell and gene therapies  (also known as Advanced Therapy Medicinal Products (ATMPs)) have revolutionised the treatment of many debilitating and life-threatening conditions.

In many cases, these therapies can target the underlying cause of a disease and can be given as a single one-off treatment offering life-long benefits to patients.

Simply put, advanced therapies are at the cutting edge of medicine.

However, such innovation brings with it additional regulatory requirements, particularly around safety, including:

  • long-term safety,
  • shedding,
  • immunogenicity,
  • germ-line transmission, and
  • environmental risk.

We have expertise in navigating the special considerations required to bring ATMPs/cell and gene therapies to market, including taking scientific advice to understand regulatory requirements around some of the specific challenges.

We are well-versed in specific initiatives that may be applicable to your advanced therapy—initiatives such as the European Medicine Agency’s Priority Medicines Scheme (PRIME); or the UK’s Innovative Licensing and Access Pathway (ILAP).

You may be developing an advanced therapy for an ultra-rare disease, in which case, it may be feasible to obtain marketing under exceptional circumstances approval. We can advise the best route to approval for your unique product and development programme.

Questions  you may have if you are developing an advanced therapy

  • What are the regulatory pathways for developing biologics/ATMPs?
  • Are there specific regulations and guidance documents for biologics/ATMPs? Particularly cell therapies, gene therapies or tissue-engineered products?
  • How can we optimise your regulatory strategy to meet the unique challenges associated with developing biologics/ATMPs?
  • What regulatory schemes are available for developing advanced cell and gene therapies (PRIME, ILAP, Breakthrough Designation)?
  • What additional requirements are we held to when developing advanced therapies?
  • Have we mitigated the risks of potential off-target effects?
  • What are the most appropriate clinical trials designs for ATMPs/biologics, given the personalised nature of these medicines?
  • What are the genetically modified organism requirements for genetically modified cells?
  • What are the environmental risk assessment requirements for genetically modified cells?
  • What are the regulatory requirements for tissue-engineered products?
  • How do we navigate the regulatory requirements for biotechnology-derived therapeutics?
  • How do clinical trials differ for ATMPs, biologics and cell and gene therapies?