RNA Delivery May Bring Stronger Vaccines

With science and technology advancing every day, new discoveries are also being made in the medicine world. Medical Scientists are working on new drugs that are more powerful and act faster in treating the same diseases. Diseases that were once incurable can now be prevented with a vaccine, and in case caught can be treated with a few tablets or injections. 

One of these remarkable discoveries that scientists believe holds great potential are vaccines made from RNA. These drugs can be used to treat cancer and also for the prevention of many infectious diseases. Because of their promising results, biotech companies are conducting more research on such vaccines, and a few of them have even gone through clinical trials.

However, they are facing some issues. One of these issues is that they must make sure the RNA gets into the right immune cells, and once it gets there, it must also produce the right amount of encoded protein. Another challenge is that the vaccine must be strong enough to stimulate a reaction big enough that wipes out all the relevant microorganisms causing the disease. 

Chemical Engineers from MIT have developed a series of lipid nano-particles that deliver such vaccines. In a study performed on mice, their newly developed RNA vaccine successfully managed to inhibit the growth of tumors. This study was performed on two different mice with melanoma, and in both cases, the growth of the tumor was slowed down, and the rate of survival increased.

An associate professor at MIT, Daniel Anderson, spoke about their study, saying that one of the discoveries of their study is that we can make RNA delivery lipids that can even be used to activate the immune system.

Traditional vaccines are made up of infectious microbes in weakened forms. The scientists then came up with the idea of making vaccines using DNA that can encode microbial proteins. However, the results of this study were not as promising as that of RNA.. 

Anderson, with his team, developed many lipid nanoparticles that protect and deliver RNA to its desired destination. Their aim is to develop carriers.

Do you think RNA will be successful in treating patients?

Can We Stop Infected HIV Cells from Replicating?

Dr. Tariq Rana and his colleagues discover a clue in stopping HIV cells from reproducing. A critical cellular player that controls HIV reproduction may be the key.

What is HIV? – Role of CD4 Cells

HIV or Human Immunodeficiency Virus is a virus that damages the immune system of the affected person and if left untreated, can lead to AIDS. HIV kills CD4 cells, also known as T cells or immune cells. 

 T cells are of crucial significance for the immune system as they are at the core of the system that modifies the immune response towards specific diseases. They can be considered as an infantry unit of your body that looks out for any invader and kills it. Low CD4 cells mean a few infantry soldiers who would make it difficult for them to defeat the invader or the disease. 

How is HIV treated?

HIV is treated by a lifelong process of medication, an antiretroviral treatment that stops HIV cells from replicating. Medication includes a combination of three drugs taken in one medicine. It reduces the amount of HIV cells in the blood of the affected person. 

The treatment is highly effective as it reduces the HIV cell to a point where it is undetectable, and HIV cannot be transmitted to another person. But the treatment has to go on for the rest of the affected person’s life; otherwise, HIV would start replicating. As soon as the treatment stops, HIV will begin replicating at a much faster pace. As of yet, HIV cannot be cured. It remains active, even though at a very negligible level, during the treatment. 

The Critical Cellular Player

The identified cellular player controlling the HIV reproduction in the immune cells discovered by Dr. Tariq is an RNA molecule dubbed as HEALS. The team adopted genetic sequencing to discover it. The research concluded that if HEALS is deleted from the body of the person, HIV reservoirs hiding in the body will be eliminated, which means that even without the antiretroviral treatment, HIV would not replicate. 

Rana reported the presence of long noncoding RNA in HIV cells, and it was discovered that genes could be turned on or off, in other words, genes can be controlled by the help of this noncoding RNA. By modifying this noncoding RNA, HIV can be prevented from resurrection in immune cells. 

Additional research would be conducted on animals, as Dr. Tariq says that the most exciting part of this discovery has not been seen before. Adding more, he said that this finding is a potential therapeutic target and could be exploited like that. 

According to a survey conducted by UNAIDS in 2017, approximately 36.9 million people in the world are HIV positive. With the help of modern research and antiretroviral treatment, very few people face the development of AIDS.

Genentech Signs Billion Dollar Deal with Sosei Heptares and Skyhawk

Genentech Inks Deal With Skyhawk And Sosei

Introduction

Genentech and Convelo Therapeutics collaborated to accelerate their discovery of the drugs to deal with different targets, such as the patients who are suffering from neurological disorders like multiple sclerosis (MS). The two companies decided to work together to discover the therapies for MS as well as other myelin disorders. According to the deal, Convelo will receive an undisclosed upfront payment and also some support during the search from Genentech. 

Since the financial terms of Convelo were not disclosed, Genentech decided to enter into another deal whose worth is $1 billion today. The deal was between Genentech and Japan-based Sosei group. They joined hands in the discovery of novel medicines that would modulate G Protein-Coupled Receptor GPCR targets.

Genentech inks discovery deals with Skyhawk, Convelo

Genentech made a deal of $100 million ($1 billion) biobucks with G Protein-Coupled Receptor (GPCR) specialist Sosei Heptares and under this deal; both the companies will work on new meds that modulate GPCR targets. According to Nature, it is believed that between one third and one-half of all marketed drugs act by binding to GPCRs. If this is true, then it completely explains why Genentech was interested in this target.

They decided to focus on two things; one focused on cancer and neurodegenerative diseases, and the other focused on neurological disorders.

For this purpose, Sosei Heptares will combine its GPCR focused structure bases drug design with Genentech’s experiment and Skyhawk will use its Sky STAR technology to find out about small molecules that can modulate RNA splicing and treat numerous oncology and neurological disorders. 

Moreover, the good news is that Genentech is now going to discover both oncology and neurodegeneration and promises to develop market drugs for multiple diseases. In exchange, it only demands the promise of future payments and royalties. 

This agreement proves to be very helpful and exclusive for Genentech all over the world, as it gets the license to develop potential therapeutics that is directed to multiple targets. On the other hand, Skyhawk receives an upfront payment and gets the eligibility to receive all the future payments and royalties. This agreement also says that, if anything happens to clinical development or commercialization, Genentech would be held responsible for that. 

The Genentech collaboration comes to two months after Skyhawk teamed up with Takeda Pharmaceutical and after a year, it penned $60 million with Celgene, and now they could license up to five new drug candidates from the deal. 

As for the deal with Convelo, both Genentech and Convelo will work on new therapies for a better treatment that might revive fatty substances which not only protects but also gives a covering on nerve fibers that appears just like some coating. 

According to James Sabry,

“We believe that GPCRs are that class which plays a great role to cure many diseases. Thanks to Sosei Heptares, because of him, there are more and more drugs being discovered every year, his outstanding capabilities accelerated the discovery of drugs.”

Mallinckrodt and Silence Therapeutics Partner up for RNAi

At first, you need to have a little introduction to these two companies. They both are pharmaceuticals. They provide medicines and research related to medicine. What are they and what do they do? Here’s the introduction.

Mallinckrodt

An Irish–tax registered manufacturer, known as Mallinckrodt Pharmaceuticals of specialty pharmaceuticals, generic drugs, and imaging agent in 2017 generated 90% of sales from the U.S. healthcare system. With such a large network, it is one of the finest and leading pharmaceuticals.

Silence Therapeutics

A biotechnology company that develops therapeutic gene technology based on RNA interference. They are responsible and have major involvement in developmental research of targeted RNAi therapeutics for the treatment of serious diseases.

What is RNAi?

RNAi or RNA Interference is a biological process in our internal body. RNA interferes with RNA to cut it down from double-stranded RNA to single and then to further more pieces. This process inhibits gene expression or translation by targeting RNA and neutralizing it. 

Mallinckrodt and Silence Therapeutics Partnership

Mallinckrodt is the finest and leading pharmaceutical you can find. With such a vast network, it has the power to change, the power to reach out and help maximum. The one problem they were facing was lack of research and information about RNAi. Silence Therapeutics had all the knowledge of RNAi. With that knowledge, you could even treat deadly cancerous diseases. You may have guessed it until now.

One of the ten principles of microeconomics says Trade can make everyone better off. So, Mallinckrodt holding power to reach out and change and Silence Therapeutics with the information made a deal and became partners.

There were some noticeable points made at the time of their partnership. Some of them are mentioned below.

•    This partnership will allow Silence’s platform in the field of RNAi technology to advance. With more advancement, new ways would come out to solve deadly diseases.

•    The agreement provides Mallinckrodt with an exclusive worldwide license for one preclinical asset that targets a specific protein in the complement pathway, C3 (SLN500), and an option for up to two additional assets with different complement protein targets

•    $20 million upfront payment, long-term development, commercial milestones, and royalties on net sales will be received by Silence.

This collaboration will allow both companies to develop an RNAi drug. This drug would be a group of proteins that are involved in the immune system to fight different diseases. These proteins play an important role in the development of inflammation.

These proteins are also considered to contribute to the pathogenesis of many diseases, including autoimmune diseases. This would change the medical world and show it a better path to find more ways to fight deadly diseases.

Not only will this partnership help in the medical field but would also leave an impact on other companies to indulge themselves in these healthy partnerships to produce the best possible outcome. The business world is a dirty place, and it needs to have examples like these.