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BETA-BET: Pioneering Gene-Editing Initiative Targets Thalassemia’s Genetic Core

Introduction

A groundbreaking project at the Cyprus Institute of Neurology and Genetics (CING) is set to transform the treatment landscape for beta-thalassemia, the most common inherited blood disorder in Cyprus. The initiative aims to deliver a one-time, curative therapy by leveraging advanced gene-editing technologies.

Path to Breakthrough

The Molecular Genetics Thalassaemia Department (MGTD) has announced the launch of the BETA-BET: Targeted Base Editing for Beta Thalassemia project. This ambitious program focuses on addressing the HBBIVSI-110 mutation, the primary genetic culprit behind the condition on the island. Beta-thalassemia arises from abnormalities in the β-globin gene (HBB), which plays a vital role in oxygen transportation through haemoglobin. Traditionally, patients have depended on lifelong blood transfusions to manage chronic anemia, a burden that this new therapy seeks to eliminate.

Expanding the Scope of Base Editing

The BETA-BET project, funded with a substantial budget of €198.83 million by the Research and Innovation Foundation (RIF) along with co-funding from the European Union and the Republic of Cyprus, pioneers the use of “base editing” technology. This method, comparable to a precise biological pencil and eraser, corrects single “letter” errors in the DNA sequence without inducing double-stranded breaks. An earlier preclinical study—conducted in collaboration with leading institutions across Greece and Germany—demonstrated the potential of these tools to effectively restore the production of functional red blood cells in individuals homozygous for the HBBIVSI-110 mutation.

Innovative Delivery System

Beyond expanding the patient group to include compound heterozygotes, whose prevalence significantly exceeds that of homozygotes in many regions, the project introduces a revolutionary delivery mechanism. Engineered virus-like particles (eVLPs) are being developed to function as microscopic delivery drones, transporting gene-editing complexes directly to blood-forming stem cells. This strategy could simplify treatment administration to a single injection, thereby eliminating the current, complicated process of ex vivo stem cell manipulation.

Collaborative Leadership and Global Partnerships

Under the expert guidance of Dr Petros Patsali, Associate Scientist at MGTD, and a team of renowned researchers, the project draws on a network of national and international partners. Esteemed collaborators include institutions such as George Papanikolaou Hospital, the Aristotle University of Thessaloniki, and the University of Freiburg, among others. These partnerships not only bolster the project’s scientific rigor but also enhance its global relevance, particularly in high-incidence areas like Greece and Egypt.

Conclusion

By extending the precision of base editing to a wider patient demographic and integrating novel delivery systems, the BETA-BET initiative represents a significant advance in gene therapy. This innovative approach could eventually provide a transformative, one-time treatment for thousands of patients, marking a milestone in the pursuit of a definitive cure for thalassemia.

Copyright Law Struggles To Keep Up With AI Training

Courts Are Still Applying Old Copyright Rules To AI

AI companies train models on enormous amounts of published material, including books, articles and academic research. Whether using that content without authors’ permission violates copyright law remains unresolved.

Much of the debate centres on fair use, which allows copyrighted material to be used without permission in certain circumstances. Courts consider factors such as the purpose of the use, how much material was involved and its impact on the original market.

Anthropic Case Sets An Important Precedent

A major case involving Anthropic and a group of authors provided one of the clearest rulings so far. Judge William Alsup found that using copyrighted books to train AI models was lawful, comparing the process to people reading and studying literature before creating something new.

Anthropic was nevertheless ordered to pay $1.5 billion in a settlement. The penalty concerned books the company had obtained from illegal online libraries rather than the AI training itself.

For AI companies, that distinction could prove significant because it separates studying copyrighted material from directly copying it.

Competition Could Be The Key Issue

A case involving Thomson Reuters and Ross Intelligence offers a different perspective. A court ruled that Ross could not claim fair use after using Reuters’ copyrighted material to develop a competing AI-powered legal research platform.

The decision suggests courts may be less willing to consider AI training fair use when copyrighted content is used to build a product that directly competes with the original.

For authors, an unresolved question is whether AI-generated content should be considered competition for the works used to train these models.

The Law Has Yet To Catch Up

US copyright law predates generative AI by decades, leaving courts to apply old principles to new technology. Questions also remain over copyright protection for AI-generated works. In Thaler v. Perlmutter, a court ruled that material created entirely by AI cannot receive copyright protection.

Major AI companies remain involved in copyright litigation, and different courts could reach different conclusions. For now, there is no universal rule: the legality of AI training will depend on the circumstances of each case and how courts ultimately interpret copyright and fair use.

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