The destruction results in impaired insulin production and subsequently dysregulated glucose levels, causing hallmark symptoms like increased thirst, frequent urination, and weight loss. Beta cell regeneration is hindered, exacerbating T1D progression. While standard treatments like pancreas or beta cell transplant and maintenance therapy exist, they face supply limitations and necessitate constant blood glucose monitoring. Gene therapy offers a promising avenue for addressing T1D's underlying causes.
In gene therapy, diseased cells are modified through gene introduction, replacement, or inactivation to potentially treat or cure them. In a 2018 study, scientists successfully reprogrammed pancreatic alpha cells into beta cell-like functionality using adeno-associated vectors (AAVs). This approach facilitated beta cell proliferation, maturation, and function, resulting in normalized blood sugar levels in mice for four months without immunosuppressive drugs. While not offering a one-time cure, gene editing presents a potential solution by reprogramming the body's DNA to avoid diseases like T1D altogether.
Strategic Moves by Pharma and Biotech Players
Vertex made significant moves in 2022 and 2024 to corner the potential T1D gene therapy market. They acquired ViaCyte for $320 million to expedite VX-880 development, licensed CRISPR Therapeutics' gene editing technology for the development of hypoimmune cell therapies, and obtained an exclusive license to C-Stem, TreeFrog’s cell manufacturing technology, to optimize the production of T1D cell therapies.
Eli Lilly acquired Sigilon Therapeutics in 2023 to gain access to its experimental cell therapies, and AstraZeneca signed a $85 million deal with Quell Therapeutics for potential candidates including a “one and done” cure for T1D.
Code Bio raised $75 million to reinvent genetic medicine for rare (Duchenne muscular dystrophy) and not-so-rare (T1D) diseases.
Regulatory Push
On the regulatory front, both the Food and Drug Agency (FDA) and European Medicines Agency (EMA) have streamlined pathways to expedite gene therapy development and approval. FDA grants expedited designations like breakthrough therapy and fast track status, exemplified by CellTrans' Lantidra, the first cellular therapy for T1D. Moreover, the landmark approval of Vertex's CRISPR-based drug highlights FDA's commitment to furthering transformative therapies, signifying a new era in regulatory support for innovative treatments in the field of T1D.
The adaptive pathways approach, adopted by EMA, aims to expedite the delivery of promising therapies to patients by facilitating iterative development with real-world evidence collection.
Despite significant advancements, the field faces challenges including immune response complexity, long-term safety and efficacy concerns, technical hurdles in gene delivery and editing, inflated costs, and ethical considerations.
Future Prospects
The cell and gene therapy landscape is rapidly evolving, accelerated by substantial investments, novel technology, and strategic partnerships among major players. Advances in enhancing specificity and survivability of regulatory T cells to suppress autoimmune responses promise a more targeted treatment approach. The convergence of innovative technology, regulatory support, and favorable market conditions are synergistically driving gene therapy beyond its origins in rare diseases toward broader mainstream acceptance.
