From Promise to Patients: Accelerating CRISPR Medicine in Europe

“When someone breaks a bone, effective treatment is available at practically any hospital. When someone has a ‘broken’ gene, a cure may be possible, but treatment is simply not available. Given today’s progress in CRISPR medicine, this is increasingly difficult to justify.”Prof. Rob Wolthuis, Department of Human Genetics, Amsterdam UMC

»Given the potential of CRISPR highlighted in popular media, people come to our clinic with hopeful determination. More and more, visitors walk into our consultation rooms asking why gene-editing treatments are still not available. Parents of children with progressive genetic diseases, in particular, want to know whether gene editing could help – if not now, then perhaps one day for younger family members. They ask what we, as an academic centre, are waiting for before CRISPR-based care can be offered, and what timelines and limitations they should realistically expect.«

Prof. Wolthuis anticipates that public expectations around CRISPR medicine will only continue to rise. »It is very difficult for families to hear that CRISPR medicines are still mostly out of reach – and also unsettling for us, as consultants, to see that while the technology is essentially ready to help more patients, the path to care is not.«

“Some of the biggest barriers for CRISPR medicines are not technical in nature anymore. They are organizational, financial, legal, and regulatory.”Dr. Daniël Warmerdam, Department of Human Genetics, Amsterdam UMC.

There is, however, an implementation route that allows clinics to treat individual patients with experimental therapies on a non-routine basis. The Hospital Exemption – which predates CRISPR – provides a legal pathway for personalized, hospital-prepared advanced therapies administered under the direct supervision of a physician when no approved treatment option exists. It is specifically designed for innovative therapies (so called ATMPs), such as gene and cell therapies. Yet not all CRISPR-based therapeutic applications will necessarily fall within the ATMP classification. »Approaches based on in vivo editing for example, may not be classified as ATMPs under the current product-based definition, meaning they cannot be offered under the Hospital Exemption. This product-type–driven classification, rather than an outcome-based model, adds another layer of complexity for personalized CRISPR applications. Even within the ATMP category, the application of the Hospital Exemption differs significantly across EU Member States, due to divergent national rules and reimbursement decisions (4). In addition, reporting of patient outcomes – both within and outside clinical trials – is often inconsistent and not always published, limiting broader learning and slowing the development of shared clinical evidence. Europe must define a clear regulatory and financial path for meaningful CRISPR care to emerge,« says Dr. Warmerdam.

The Bottleneck Between European Clinics: Data That Can’t Cross Borders

“We need to let go of the idea that CRISPR therapies are a traditional medicinal product, they are a method – just like surgical procedures – and our regulatory, legal, and clinical frameworks must adapt accordingly.”Dr. Daniël Warmerdam, founding partner and steering committee member of EGMEDC.

Prof. Rob Wolthuis envisions an approach that places academic medical centres at the heart of innovation. »We have an exceptional opportunity here because university hospitals are uniquely positioned to drive the next phase of CRISPR medicine,« says Prof. Wolthuis. »Academic medical CRISPR centres can combine direct patient care with deep scientific and genetic expertise, and can potentially provide personalized services at direct cost. That mix may be the key to bringing individualized gene-editing therapies into the clinic.«

The idea is straightforward: combine the programmability of CRISPR with clinical-grade building blocks to create what is essentially a genetic form of surgery. In this model, clinical specialists design a curative and safe gene editing plan, while commercial partners may provide standardized, high-quality components such as CRISPR enzyme-encoding mRNAs, in vivo delivery vehicles, or custom clinical-grade guide RNAs, similar to companies supplying surgical tools or custom titanium plates to stabilize bone fractures. The guide-RNA design, safety assessment, and GMP components converge in a clinical-grade platform for CRISPR medicines that can evolve into an accredited pipeline where personalized adaptations are within regulations.

Turning Science into Treatment: A European Genomic Medicine Consortium

Given Europe’s fragmented landscape, developing and delivering personalized CRISPR therapies for rare-disease patients remains an enormous and deeply complex challenge for academic centres. »We not only need to work across national borders to evolve current practices but also push meaningful regulatory changes to harness the power of CRISPR across disease boundaries. Take the therapy given to baby KJ to repair his CPS1 gene deficiency: a highly similar approach could be used for other urea-cycle disorders – and even other genetic liver-associated diseases – simply by tailoring the guide-RNA and editing enzymes to each patient. What we need in Europe is an integrated environment that aligns scientific progress, responsible regulatory freedom, balanced safety oversight, and real economic support for CRISPR medicine,« says Prof. Rob Wolthuis.

Dr. Daniël Warmerdam agrees:»Progress will depend on a regulatory and financial environment that allows clinicians to conscientiously deliver CRISPR-based therapies within a transparent European network-one that promotes the dissemination of innovations and the sharing of treatment outcomes. We can only evaluate the benefits of these genomic medicines once they are put into practice, starting with severe conditions that have no effective therapies.«

Building on this perspective, a European alliance is now emerging. The non-profit European Genomic Medicine Consortium (EGMEDC) aims to create an actionable, lean, cross-border network for experimental genomic medicine implementation. By communicating openly through CMN’s established channels and social media, EGMEDC aims to inspire, challenge, and energize Europe’s fragmented landscape – and to identify and implement practical solutions to the roadblocks that hold back progress today. EGMEDC invites partners to join this effort and accelerate the translation of CRISPR and other genomic medicines into real clinical benefit. In the European spirit of ‘all for one, and one for all, (5)’ we can bring vital treatments to people living with rare genetic diseases and pave the way for broad societal benefit through genomic medicine.

“Only by building this community of shared knowledge and responsibility can Europe ensure that when a patient walks in with a broken gene, their cure is not a distant dream but a genuine possibility.”Prof. Rob Wolthuis, EGMEDC founding partner and steering committee member.