바이오테크로 암과 싸우다 – mRNA 백신이 왜 큰 도약인지

mRNA 혁명

When the COVID-19 pandemic struck, the world’s medical community initially prepared for a disaster of the scale of the Spanish Flu, which struck during WW1 and killed 50 million people (from a much smaller global population). Instead, “only” 7 million people are considered to have died from Covid.

mRNA 백신은 당시 새로운 기술이었으며 팬데믹 동안/이후에 매우 빠르게 배치되었습니다. 기존 백신 기술과 비교했을 때 mRNA 백신이 독특했던 이유는 개발의 단순성에 있습니다. 기존 백신은 바이러스 입자를 불활성화하거나 약화시키는 정확한 프로토콜을 찾고, 바이러스가 어떻게 병원성을 갖는지와 면역 체계가 이를 어떻게 인식하는지를 파악한 뒤, 실제로 효과가 있는지 확인하기 위해 수개월 또는 수년간 테스트해야 했습니다.

mRNA 백신은 대신 바이러스 게놈을 직접 살펴볼 수 있었으며, 이는 Covid-19가 발견된 첫 주에 확보된 데이터였습니다. 그리고 바이러스 중 하나에 해당하는 mRNA 서열을 만들어 환자의 몸이 해당 단백질을 생산하도록 합니다.

이렇게 하면 면역 체계가 바이러스 단백질에 직접 반응하는 방법을 배우게 되며, 실제 바이러스에 노출되었을 때 즉시 활성화될 준비가 됩니다.

출처: ESCO Life Sciences

바이러스 발견부터 백신 제작까지의 과정이 몇 주에 불과하고 대부분이 유전체 데이터에 의해 주도되기 때문에, 이는 고전적인 생물학 연구보다 “코드를 작성하는” 의학에 비유됩니다.

This creates hope that other diseases that have been resistant to vaccination so far could be prevented with mRNA vaccines. For example:

• Moderna (MRNA )는 mRNA vaccines for RSV, EBV (mononucleosis), HSV (herpex), VZV (varicella), HIV, norovirus, Lyme disease, Zica, Nipah, and mPox vaccines를 개발하고 있습니다
• BioNTech (BNTX )는 HSV, tuberculosis, malaria, mPOX, and shingles에 대한 mRNA 백신을 개발 중입니다
• Curevac (CVAC )도 bird flu, Lassa yellow fever, RSV, rabies, malaria, and rotavirus에 대한 mRNA 백신을 연구하고 있습니다.

감염성 질환을 넘어

As the mRNA vaccine works by training the immune system to focus on a given target, determined by its genetic sequence, it can theoretically be trained to attack any type of specific protein, not just infectious diseases.

우리는 또한 암 세포가 건강한 세포에는 없는 특정 마커를 표시한다는 것을 알고 있습니다. 이것이 바로 CAR-T 치료법 및 단클론 항체와 같은 모든 암 면역 치료의 기반이며, 우리는 이를 “단클론 항체: 원조 정밀 치료”에서 논의했습니다.

문제는 종종 이러한 암 세포를 감지하도록 면역 체계를 훈련시키는 것이 비싸고 실험실에서 항체나 면역 세포를 맞춤 제작하는 것이 규모를 확대하기 어렵다는 점입니다.

This is exactly what mRNA-based cancer therapy is looking to achieve. And progress is being made quickly.

mRNA 폐암 임상 시험

At the end of 2024년 8월, mRNA vaccine leader BioNTech (producer of the “Pfizer vaccine”) announced that it started global clinical trials in 7 countries and 34 research sites for a mRNA-based lung cancer vaccine, called BNT116.

The focus will be non-small cell lung cancer (NSCLC). It will involve around 130 patients, who will receive BNT116 in combination with immunotherapy, with the goal to boost the efficiency of the treatment.

The trial will enroll patients at different stages of NSCLC, from early-stage disease before surgery or radiotherapy (Stage 2 and 3) to late-stage disease (Stage 4) or recurrent cancer.

It is a more complex therapy than mRNA vaccines like the Covid-19 vaccines, as it includes six consecutive injections five minutes apart over 30 minutes, with each jab containing different RNA strands. The patient will then go on to get the vaccine every week for six consecutive weeks, and then every three weeks for 54 weeks.

재발 감소

A key expected effect of the mRNA treatment is to reduce the re-occurrence of cancer once the treatment is done, as the effects are expected to be much more lasting than with classical immunotherapies.

This is because the “trained” immune cells will stay active and vigilant against the cancer market, the same way they would be against the risk of a future infectious disease attacking the body.

This also means that while the treatment is not preventing cancer, if it works, it will truly deserve the “vaccine” qualification, at least against cancer re-occurrence.

기타 mRNA 활용

Because of its versatility in leveraging the immune system, mRNA technology could be used for a variety of ailments beyond infectious diseases and cancers.

Among the various possible applications, we can mention:

• 희귀 질환 및 유전 질환: including cystic fibrosis and phenylketonuria.
• 진단: as cancer cells express different protein profiles than normal ones, their mRNA coding for these proteins also differs. This can open the way for cancer detection through a technique called 액체 생검.
  • Such diagnostic tools will allow for early cancer detection, dramatically increasing the survival rate.
• 심근경색 및 손상된 장기 치유: 펜 메디신 연구원들은 mRNA를 사용하고 있습니다 to modify liver genes and reduce heart attack risks. They are also working on a cure for fibrosis in the heart tissues, a major cause of heart failure.
• 보다 안전한 유전자 치료: While most gene therapies look to insert DNA into cells or to modify DNA using CRISPR technology, this can have unintended consequences as the insertion is permanent.
  • For more transient effects that still rely on gene therapy, injection of mRNA into cells can be used, although this technique is still very recent, and appropriate mRNA delivery systems have only been developed in the last few years.
• 자가면역 질환: mRNA vaccine can be used to reduce instead of stimulate the immune system response, which is required in auto-immune diseases, where the body attacks healthy cells.
  • This can be achieved by inducing the proliferation of Regulatory T cells (Treg), which reduce the intensity of the erroneous immune reaction.

mRNA 위험성

After the quick emergency unrolling of the mRNA vaccine during the pandemic, many feared that it was actually causing plenty of unintended side effects, like heart inflammation (myocarditis).

Combined with the politicization of the vaccines and the chaos of lockdowns and the pandemic, this has led to a lot of confusion.

A few years later, we are now starting to get strong data. While rare, the cases of myocarditis are real, and according to the CDC, “have most frequently been seen in adolescent and young adult males within 7 days after receiving the second dose of an mRNA COVID-19 vaccine”.

Further research from 2024년 9월 indicates that the interval between the vaccine and booster shot might be an important factor:

“We show that longer intervals between each consecutive dose, including booster, may decrease the occurrence of vaccine-associated myocarditis by up to a factor of 4, especially under age 50. These results suggest that a minimum 6-month interval might be required when scheduling additional booster vaccination.”

Influence of mRNA Covid-19 vaccine dosing interval on the risk of myocarditis

– Nature Communication

So overall, mRNA, like most treatments, is never 100% without side effects.

However, when it comes to new applications, like aggressive and re-occurring lung cancers, the risk of side effects can likely be negligible compared to the increased survival rate.

mRNA 백신에 투자하기

mRNA has become, in large part due to the pandemic, a darling of investors in 2020-2022. The necessary cooling down of mRNA vaccine sales post-pandemic has caused the prices of many mRNA-related stocks to decline as a result.

This does not change the fact that technology is remarkably powerful in preventing diseases, from infectious diseases to cancer, and maybe rare diseases, auto-immune syndromes, etc. And the company’s expertise in mRNA vaccines gives it a serious headstart in bringing this tech to new fields of medicine.

You can invest in mRNA companies through many brokers, and you can find here, on securities.io, our recommendations for the best brokers in 미국, 캐나다, 호주, 영국, 및 기타 여러 국가.

If you are not interested in solely mRNA companies, you can also look into biotech ETFs like WisdomTree BioRevolution UCITS ETF (WBIO), VanEck Biotech ETF (BBH), or First Trust NYSE Arca Biotechnology Index Fund (FBT), which will provide more diversified exposure to capitalize on the growing biotech economy.

mRNA 솔루션을 제공하는 기업

One of the leading companies in mRNA cancer therapies is BioNTech, which is building from its success in developing the mRNA vaccine commercialized by Pfizer.

Cancer

BioNTech currently has 11 different candidates in cancer treatments, covering cancer in the ovaries, prostate, intestines, skin, head, neck, and multiple solid tumors.

Most oncology treatment clinical trials are in phase 1/2, with already three candidates in phase III.

출처: BioNTech

In total, the company has 21 clinical programs in oncology. This includes not only mRNA, but also small molecules, Immuno-Oncology agent (IO), and Antibody-drug-conjugates (ADCs).

The first oncology product is expected to be potentially launched in 2026.

출처: BioNTech

Infectious Diseases

BioNTech also stayed the leader in COVID-19 vaccines, with >50% market share, and upcoming combination respiratory vaccines (Covid + Flu/Influenza) in late 2025 or 2026 if approved.

Regarding infectious diseases, BioNtech is pursuing vaccines for shingles, Herpex, tuberculosis, malaria and Mpox. Among those, the diseases affecting the most people are Herpex viruses (3.7 billion people infected), malaria (249 million), and tuberculosis (10.6 million).

출처: BioNTech

AI

BioNTech is also very active in the AI-Biotech field, with the creation in 2020 and full acquisition in 2023 of the company InstaDeep AI.

출처: BioNTech

InstaDeep is the “first AI Immunotherapy Platform”, using LLM technology on DNA and protein sequences, AI vision for histology (tissues under a microscope) analysis, and AI agent for lab automation and quality control.

The goal is to deploy AI across the entire R&D pipeline.

It uses a supercomputing cluster with 224 Nvidia H100 GPUs and 86,000 CPU cores, with 0.5 ExaFLOPS, making it into the top 100 worldwide. InstaDeep’s genomics AI models are among the most downloaded, illustrating how they are “state-of-the-art” in this field.

Financials

The money from the pandemic gave the company a very strong position, with €16,9B in total available cash by mid-2024. In 2023, Covid-19 vaccines still generated €3.8B in revenues, for a gross profit of €3.2B.

This is likely to slow down in 2024, but overall, the company has very strong finances for what is still ultimately an early-stage biotech startup with only one product commercialized.