2023-03-02| In-DepthSpecial

A New Phase in Drug Development: Programming the Genome

by Elena Chen
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The International Center for Genetic Disease (iCGD) is a platform that analyzes patients and healthy subjects from different parts of the world for research into the causes and consequences, prevention, and treatment of disease. On February 21, the iCGD invited Noubar Afeyan, Ph.D., Founder and CEO of Flagship Pioneering and Co-founder and Board Chairman of Moderna, to present “Programmable Genomic Medicines & Platforms” as part of their keynote series.

Genome Programming and the Founding of Moderna

Flagship Pioneering is an organization based in Cambridge, Massachusetts, and has been around for about 23 years. Composed of 350 core members, the organization invents platforms to form the basis of startup companies, which are nurtured with the eventual goal of spinning out as standalone companies. 

Afeyan highlighted that many different products are produced from a platform. According to Afeyan, a “platform” is a common way to produce different products, like Apple or Microsoft operating systems, which in turn create other products. In biology, it may also follow similar models to produce different corresponding products, new forms of medicine, and new technologies are used to derive diversified products. 

Take Flagship Pioneering as an example. In the past ten years, research on nucleic acid has developed a new generation of CAR-T therapies that can be effectively delivered to many disease areas. At the same time, the company is also actively participating in developing artificial intelligence and promoting its application in life sciences.

For gene-edited therapeutics, protein-coding is essentially a simplification of the central principle of DNA-compiling RNA. However, what would be the consequences if the wrong code is executed, or the correct code or mechanism was executed incorrectly? With that question in mind, Dr. Noubar Afeyan and his partners founded Moderna, hoping to analyze and find answers by creating biological platforms.

Moderna today is not only working on developing and supplying vaccines against COVID-19 but is also running 48 programs in development, preclinical, and clinical stages. Currently, they have four different modalities in Phase 3 trials and many more in advanced Phase 2 trials. Besides cutting across many disease areas, including oncology and autoimmune diseases, Moderna also participates in the development of vaccines, replacement therapies, etc.

Noubar Afeyan, PhD, Founder and CEO of Flagship Pioneering and Co-founder and Board Chairman of Moderna

Related Article: Moving Beyond COVID With mRNA Technology

The Pandemic has Forced Accelerated Vaccine Productivity

From receiving the public sequences of the coronavirus SARS-CoV2 on January 11, 2020, an optimized sequence quickly progressed into human trials in partnership with NIH. A mere 44 days later, the first Moderna COVID-19 vaccine was tested in an extensive Phase 3 study, 

Afeyan believes the key of success of this journey were not one but many. First, Moderna has engaged in mRNA research and development for many years; their familiarity with the spike protein was far ahead of other companies. 

Secondly, mRNA is an information molecule, and Moderna had already built the necessary underlying platform that could be deployed for any task including the latest emergency. Furthermore, the company’s previous research on the MERS virus which shares many similarities to SARS-CoV-2, made it easier and faster to scale up vaccine production. They have a manufacturing facility, albeit one that was making cancer vaccines, which was converted quickly to accommodate COVID-19 vaccine production. Lastly, the massive sacrifice of hundreds and hundreds of scientists working 7 days a week was an important factor in the success of the mission. 

Years ago, research on treating diseases might take several years. For example, hepatitis research took as long as 16 years, and the study of AIDS is still ongoing. But this time, in response to the rapid spread of the epidemic, it was necessary to find countermeasures urgently. From identifying the virus to developing a vaccine, it was completed within one year. This incident forces researchers to consider whether to go back to the long research and development process as in the past or to learn from this experience and apply it to a broader range of research fields. In the case of Flagship Pioneering, the company will focus on gene programming and editing, trying to go beyond pure mRNA drug development. 

In the following section, Afeyan focused on four companies that specialized in engineering beyond DNA, RNA, protein, and genes.

The Four Latest RNA-based Companies Will Create an Innovative Future

Tessera Therapeutics:  Over the last 4 years, 11 trillion DNA base pairs of published sequences of various life forms have been involved in a substantial search to extract features of the genetic elements and convert them into writing platforms. Tessera is pioneering Gene Writing technology to write “therapeutic messages” into the human genome, with the intent of curing diseases at their source. This technology can correct single nucleotides, delete or insert short sequences of DNA, and write entire genes into the genome, offering the potential for a new category of genetic medicines with broad applications. 

Omega Therapeutics: The first company to harness a systematic approach to use mRNA therapeutics as programmable epigenetic medicines, Omega’s platform restores aberrant gene expression to a normal range by durably tuning genes pre-transcriptionally without altering nucleic acid sequences. Omega is advancing a pipeline of candidates spanning a range of disease areas, including oncology, regenerative medicine, multigenic diseases, including immunology, and select monogenic diseases, such as alopecia. 

Laronde: A pioneer of Endless RNA (eRNA), a platform that enables persistent endogenous protein creation. This uniquely engineered RNA can be programmed to express a wide range of protein modalities. It is persistent, non-immunogenic, and allows for repeat dosing with versatile formulation and delivery. Laronde is rapidly scaling to develop multiple programs across many disease areas. 

Alltrna: Being the world’s first transfer RNA (tRNA) platform company to pioneer tRNA therapeutics to treat diseases, Alltrna has successfully applied machine learning to reveal the unique language of tRNA biology and to program tRNA medicines. This technology offers the opportunity to develop single tRNA medicines that can each treat thousands of diseases with the same genetic mutation. 

Finally, Afeyan concluded that new RNA platforms are coming out one after another. The revolution of technology may be a foreword to successful drug development that can fulfill much unmet medical need. 

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