From One Patient to a New Model of Medicine: The Rise of Custom Genetic Therapies

In early 2026, the United Kingdom initiated a new regulatory pathway allowing a small group of children with ultra-rare neurodegenerative disorders to receive individualized antisense oligonucleotide (ASO) therapies under a standardized framework.

Reported by The Economist (January 2026), this initiative marks an important shift: it evaluates not only a single drug, but the process used to create customized medicines.

While still early, this model reflects a broader transformation in genomic medicine — one moving from population-based treatments toward mutation-specific interventions.

What Is an Ultra-Rare Genetic Disorder?

Ultra-rare disorders are typically defined as affecting fewer than 1 in 50,000 individuals (European Medicines Agency, EMA).

Individually rare, these conditions collectively represent a substantial global burden:

• Approximately 300 million people worldwide live with a rare disease.
• Around 80% are genetic in origin.
  (Source: NORD – National Organization for Rare Disorders; EURORDIS; WHO)

Many ultra-rare diseases are caused by a single pathogenic mutation. The biological mechanism may be clear — yet traditional drug development models are not designed for conditions affecting only a handful of patients.

Understanding Antisense Oligonucleotides (ASOs)

The treatment platform discussed in the UK initiative relies on antisense oligonucleotides (ASOs).

To explain simply:

• DNA stores genetic instructions.
• These instructions are transcribed into messenger RNA (mRNA).
• mRNA guides protein production.
• A mutation can alter the RNA message, leading to dysfunctional proteins.

An ASO is a short, synthetic strand of nucleotides designed to bind to a specific RNA sequence.

Depending on the design, it can:

• Block production of a harmful protein
• Modify RNA splicing
• Restore more functional protein production

ASOs have already been approved for certain genetic conditions, including:

• Spinal Muscular Atrophy (nusinersen / Spinraza)
• Duchenne Muscular Dystrophy (eteplirsen)

(References: FDA; EMA; Bennett et al., Annual Review of Pharmacology and Toxicology, 2017; Crooke et al., Cell Metabolism, 2018)

ASOs are not universal therapies. They are mutation-specific and applicable only in certain genetic contexts. However, they represent one of the most adaptable RNA-targeting platforms currently in clinical use.

What Changed in the UK?

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) introduced a “master protocol” framework.

Rather than requiring a full, independent regulatory pathway for each customized molecule, regulators are evaluating:

• Manufacturing consistency
• Chemical structure and platform reliability
• Safety monitoring standards
• Pharmacokinetics (how the molecule behaves in the body)

If validated, this could allow multiple individualized ASOs to be developed under a shared, approved process.

This concept resembles platform-based approvals used in other contexts (for example, annual influenza vaccine updates).

The goal is not deregulation. It is regulatory adaptation for ultra-rare conditions where traditional randomized trials are not feasible.

(References: MHRA, 2026 announcement; The Economist, Jan 2026; Genomics England; Rare Therapies Launch Pad initiative)

The Importance of Timing

Many ultra-rare neurodegenerative diseases cause progressive and irreversible cellular damage.

Scientific literature increasingly suggests that earlier molecular intervention may offer better outcomes in some conditions (Finkel et al., NEJM, 2017; Mendell et al., NEJM, 2017).

This raises an important long-term consideration:
If individualized therapies become scalable, earlier genomic identification — including expanded newborn screening — could become more clinically meaningful.

Organizations such as Genomics England are already exploring expanded genomic sequencing programs in pediatric populations.

However, screening and treatment expansion require careful ethical, economic, and regulatory evaluation.

The Adnà Perspective: Our Role in the Ecosystem

It is essential to be precise.

Adnà is not a diagnostic laboratory.
We do not diagnose disease.
We do not provide medical interpretation.
We do not develop or administer treatments.

We operate in a complementary space — focused on accessible genetic information, education, and human-centered engagement.

Personalized medicine exists on a continuum:

• At one end: certified clinical diagnostics, regulatory oversight, and therapeutic intervention.
• At the other: curiosity-driven exploration, literacy, and informed awareness.

Adnà contributes to the second.

Genetic curiosity often precedes clinical pathways.
Accessible information can prompt informed discussions with healthcare professionals.
Understanding one’s genetic landscape may encourage responsible medical consultation.

But these steps must remain clearly separated from clinical diagnosis and treatment.

We support literacy — not substitution.

Because Your DNA Has Answers.

References

• The Economist (Jan 19 & Jan 21, 2026)
• MHRA (UK Medicines and Healthcare products Regulatory Agency) – 2026 master protocol announcement
• Genomics England – Rare Therapies Launch Pad
• NORD (National Organization for Rare Disorders)
• EURORDIS Rare Diseases Europe
• WHO – Rare Disease Global Data
• Bennett CF & Swayze EE. Annual Review of Pharmacology and Toxicology, 2017
• Crooke ST et al. Cell Metabolism, 2018
• Finkel RS et al. New England Journal of Medicine, 2017
• Mendell JR et al. New England Journal of Medicine, 2017

Important Disclaimer:

The therapies described in this article are experimental or highly specialized treatments delivered within regulated hospital environments under strict medical supervision.

Nothing in this article constitutes medical advice.

Adnà does not replace medical consultation, diagnosis, or treatment.