Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine

Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine

technology By Jan 12, 2024 No Comments

Advancing Antisense Oligonucleotides For Personalized Medicine

Given the potential of ASOs to treat rare genetic disorders, efforts to advance this technology are crucial for life sciences innovators and the patients they serve.

Optimizing ASO research and Development

The 2023 Oligonucleotide Therapeutics Society meeting in Barcelona showcased the growing interest in nucleic acid-based drugs, particularly antisense oligonucleotides (ASOs). ASO technologies hold promise for personalized treatments, especially for ultra-rare diseases which have been historically difficult to address using conventional small molecule drugs.

Demaris Mills, president at Integrated DNA Technologies, underlined the conference’s buzz around the potential of ASOs to revolutionize the treatment landscape. The FDA and EMA have approved ASO drugs, including those addressing Duchenne muscular dystrophy and spinal muscular atrophy, reflecting the tangible progress in this therapeutic class.

Enhancing Patient Identification and Target Discovery

To expedite the clinical development of ASOs for ultra-rare diseases, artificial intelligence (AI) presents an opportunity for identifying patient-specific genetic diseases and optimizing ASO therapeutics. AI can accelerate the discovery of new ASOs, analyze mutant genes, and predict the best positioning for splice-shifting oligonucleotides.

Collaboration across the pharmaceutical industry, academia, and regulatory agencies is essential to create a decentralized database of affected patients and establish coordinated approaches. Leveraging AI can streamline the discovery process and expedite the delivery of ASO therapies to those in need.

Innovations in Oligonucleotide Drug-Delivery technology

The effectiveness of ASOs has been limited by their size, often making delivery to target cells challenging. ASOs delivered intravenously may not reach vital muscles, hindering their therapeutic impact. Researchers are exploring alternatives such as cell-penetrating peptides and ligands to improve tissue targeting and enable more precise and efficient delivery to vital organs.

These innovations have the potential to transform moderately effective ASOs into life-changing therapeutics for conditions that have historically been difficult to address.

Advancements in Development and Manufacturing

The development of ASO therapeutics necessitates high throughput small-scale synthesis of candidate compounds, followed by medium-scale synthesis for testing and large-scale synthesis for the final drug. Economies of scale could help reduce costs and make the treatment of rare diseases feasible.

To overcome Manufacturing challenges, methods to modularize, parallelize, and reduce quality-control requirements are needed. Addressing these complexities can significantly reduce development costs and accelerate the availability of ASO therapies.

Regulatory Requirements for N=1 Rare Disease Treatment Compounds

As ASOs continue to gain traction in personalized medicine, the current regulatory framework must adapt to support the rapid development of new therapeutics. To address the unique nature of n-of-1 ASO therapeutics, new guidelines are essential to enable realistic budgets and expedite the regulatory approval process.

By viewing ASOs as “drug platforms” with standardized elements but allowing for variable sequences, developers can navigate the regulatory landscape more effectively. This approach could streamline safety testing before clinical trials, substantially reducing the cost of n-of-1 drug development.

Collaborative Efforts for Breakthrough Results

The success of advancing ASOs for personalized medicine hinges on collaborative efforts among researchers, drug developers, technology makers, and regulators. Given the remarkable potential of ASOs to address rare genetic disorders, these collective efforts are poised to yield groundbreaking results for both the life sciences industry and the patients it serves.

For ASOs to reach their full potential, it is imperative that all stakeholders work together to overcome challenges, accelerate research and development, and deliver personalized treatments to those in need.

Source: forbes

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