Cell and Gene Therapies
We have expertise in navigating the special considerations required to bring Advanced Therapy Medicinal Products to market.
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 lifelong benefits to patients.
Simply put, advanced therapies are at the cutting edge of medicine.
However, the question becomes, how are advanced therapies regulated?
An example of regulatory considerations for advanced therapies
Take the approval of Casgevy, (exagamglogene autotemcel) to treat Beta thalassaemia requiring transfusions and sickle cell disease, using CRISPR-Cas9 technology. Discussions on the ethics surrounding GEMPs have been ongoing for several years, with numerous GEMP products currently in development. However, the Casgevy approval could signal the start of GEMPs being approved for different illnesses sooner than we think.
Regulatory authorities discuss the potential regulatory implications for advanced therapies like GEMPS utilising zinc finger nucleases, transcription activator-like effector nucleases (TALENs), CRISPR–Cas9 editors and prime editors.
On one hand, there is hope for the potential of GEMPs to target rare diseases, particularly in instances where several mutations target a similar phenotype. Going so far as to note the potential for GEMPs to become patient-specific, using these novel therapies to edit according to an individual’s genotype. However, several developmental considerations are necessary to get to this point due to factors unique to GEMPs.
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How are genome-editing medicines shifting the regulatory landscape?
Genome-editing medicines mark a profound shift in the future regulatory landscape. This is due, in large part, to the fact that these therapies may treat certain diseases in a single dose.
How we help companies develop advanced therapies, biologics and cell and gene therapies
The innovation of advanced therapies brings with it additional regulatory requirements, particularly around safety, including:
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 Medicines 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.
Subscribe to our Monthly Regulatory Intelligence Scan
Our monthly newsletter gives you the latest European and UK regulatory intelligence. We bring you new and interesting approvals; fast-to-market applications, orphan designations; paediatric applications and therapy area-specific news.
You can unsubscribe at any time from the link in the newsletter footer. Read our Privacy Policy here.
FAQs
How are advanced therapy medicinal products (ATMPs) classified?
Gene therapy medicines: inserting recombinant genes into the body to treat diseases including genetic disorders, cancer or chronic diseases.
Somatic-cell therapy medicines: cells or tissue that have been manipulated to change their biological characteristics.
Tissue-engineered medicines: cells or tissues modified to repair, regenerate or repair human tissue.
What are the European legal frameworks for ATMPs?
There isn’t just one, but the overarching framework is Regulation (EC) No 1394/2007
What are the incentives for developing an advanced medicinal therapy product?
65% fee reduction for a request for scientific advice for ATMPs (90% for SMEs);
90% fee reduction for the certification procedure.
Access to the Innovation Task Force to work with the EMA and ATMP developers in the early stages of ATMP development.