October 5, 2022

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Top 10 RNA therapy companies to watch in 2022

Top 10 RNA therapy companies to watch in 2022



 

Top 10 RNA therapy companies to watch in 2022.

RNA is an important macromolecule in living organisms. Genetic information is transferred from DNA to RNA through transcription, and then translated into protein by RNA, which regulates various physiological activities of living organisms.

 

In 2021, the enthusiasm for financing in the field of RNA therapy is obvious to all, and new technologies and new treatments are emerging.

Looking forward to 2022, we have screened 10 RNA therapy companies worthy of attention for your reference.

 

1. Aibo Bio

  • Core technology: mRNA vaccine
  • Key Project: ARCoVaX (Clinical Phase III)
  • Trends in 2021: B rounds, C rounds and C+ rounds of financing exceed US$1.1 billion, and key projects have entered clinical phase III
  • Rating: ⭐⭐⭐⭐⭐

Aibo Bio was founded in 2019 by Dr. InBev. The company is located in Suzhou and focuses on mRNA drug research and development.

In October 2020, Aibo Bio completed a round of financing of 150 million yuan, and completed 3 rounds of financing in 2021: a round of financing of 600 million yuan in April, and a round of C financing of 720 million US dollars in August; Completed $300 million C+ round of financing, with a cumulative financing of over $1.1 billion, ranking first in global biotechnology financing in 2021.

Aibo Bio has established an mRNA and nano-delivery technology platform with independent intellectual property rights, and its product pipeline covers the fields of infectious disease prevention and tumor immunity.

The COVID-19 mRNA vaccine ARCoVaX jointly developed with the Academy of Military Medical Research of the Academy of Military Sciences and Watson Bio is the first mRNA vaccine approved for clinical trials in China, and it has entered clinical phase III.

 

2.Laronde

  • Core Technology: Circular RNA
  • Trends in 2021: Nearly $500 million in Series A and B financing
  • Rating: ⭐⭐⭐⭐⭐

Laronde was incubated and established in 2017 by Flagship Pioneering, a well-known venture capital institution. The company is located in Cambridge, USA, and focuses on the development of circular RNA therapeutics. Laronde has raised a $50 million Series A and a $440 million Series B in 2021.

The circular RNA developed by Laronde is called eRNA, which stands for endless RNA. Natural circular RNAs generally cannot be translated because they do not have a cap structure at the 5′ end, but eRNAs can initiate capless translation because they use the translation “magic weapon” of RNA viruses – the internal ribosome entry site (internal ribosome entry site). , IRES), circular RNAs with added IRES elements can initiate continuous translation.

 

Because eRNA has no free ends, it is not recognized by the innate immune system or exonuclease, and has the properties of high stability and prolonged efficacy. In addition, the expression of eRNA therapeutic proteins is modular and programmable. By changing the protein-coding cassette of eRNA, the body can produce a variety of peptides, enzymes, antibodies, channel proteins and receptors in and out of cells. protein.

 

3.Omega Therapeutics

  • Core technology: mRNA-encoded epigenetic controllers
  • Key Project: OTX-2002 (Preclinical)
  • Trends in 2021: Series C, IPO raised more than $250 million, and cooperated with Stanford University on related treatments for eye diseases
  • Rating: ⭐⭐⭐⭐⭐

Founded in 2017 and incubated by Flagship Pioneering, Omega Therapeutics is committed to regulating gene expression from an epigenetic direction. Since its establishment, Omega has received over $210 million in financing and successfully listed on Nasdaq in July 2021.

The epigenomic controller OEC developed by Omega is a modular, programmable mRNA-encoded therapy with linkers linking the DNA-binding domain and epigenetic effector proteins. The OEC is delivered intracellularly via lipid nanoparticles to function .

Top 10 RNA therapy companies to watch in 2022

At present, Omega’s pipeline has a variety of investigational therapies, targeting a variety of indications in the fields of regenerative medicine, oncology, and monogenic diseases.

Top 10 RNA therapy companies to watch in 2022

 

4.Entrada Therapeutics

  • Core technology: endosome escape carrier
  • Project Focus: ENTR-601-44 (Preclinical)
  • 2021 trends: B round, IPO nearly 300 million US dollars
  • Rating: ⭐⭐⭐⭐⭐

Founded in 2016, Entrada Therapeutics is committed to the development of intracellular delivery technology. The company is located in Boston, USA. Since its inception, Entrada has raised $175 million in financing and successfully listed on Nasdaq in October 2021.

The endosome escape vector developed by Entrada can efficiently deliver proteins, peptides and nucleic acids into cells, which is very effective for the treatment of diseases with intracellular targets.

Entrada applies endosomal escape vector technology to the delivery of antibodies, enzymes and oligonucleotides, and has a pipeline of oligonucleotide therapies targeting neuromuscular diseases.

Top 10 RNA therapy companies to watch in 2022

 

5.Shape Therapeutics

  • Core technology: RNA editing, tRNA
  • 2021 trends: $112 million in Series B financing, over $3 billion in cooperation with Roche
  • Rating: ⭐⭐⭐⭐

     

Founded in 2018, Shape Therapeutics is headquartered in Seattle, USA. The technology developed by Shape is based on the work of Dr. Prashant Mali during his postdoctoral research in Professor George Church’s laboratory.

Shape has received nearly $150 million in financing since its inception ($35.5 million in Series A, $112 million in Series B), and reached a partnership of over $3 billion with Roche in August 2021. Shape will use a proprietary platform to develop Treatments for Alzheimer’s, Parkinson’s and other rare diseases.

The technology developed by Shape has three main directions: RNAFix, RNASkip and Delivery – AAVid.

 

Top 10 RNA therapy companies to watch in 2022
RNAFix, RNASkip and Delivery – AAVid Platform (Source: Shape)

Among them, RNAfix is ​​a gRNA (guide RNA) that edits a single base by recruiting ADAR enzymes, a natural RNA editor; RNAskip is an inhibitory tRNA (transfer RNA) used to treat premature Disorders caused by stop mutations (i.e., mutations in the codon of an amino acid to a stop codon, resulting in premature termination of peptide chain synthesis) that recognizes premature stop codons, allowing translation to be read through, resulting in a fully corrected protein; Delivery – AAVid coat The Shell discovery platform produces AAV vectors with superior delivery properties that efficiently deliver RNAfix and RNAskip payloads, as well as other payloads, into specific tissues.

 

6. Ring Code Creatures

  • Core Technology: Circular RNA
  • Trends in 2021: Completion of angel round and pre-A round of financing
  • Rating: ⭐⭐⭐⭐

Circular Code Bio, located in Shanghai, is the first company in China to deploy circular RNA therapy. It was founded in 2018.

In 2021, Ringcode Bio completed an angel round of nearly 10 million US dollars and a pre-A round of financing of over 20 million US dollars. Professor Wang Zefeng, the founder of the company’s science, is currently the head of the RNA systems biology research group of the Key Laboratory of Computational Biology, Chinese Academy of Sciences. CTO Dr. Yang Yun was a postdoctoral fellow of Professor Wang Zefeng.

At this stage, Ringcode Bio will verify the effect of circular RNA in vaccine and protein replacement or overexpression in the two directions of vaccine and protein replacement therapy, and will develop different RNA drugs in parallel based on the verification results in multiple pipelines.

 

7.Strand Therapeutics

 

  • Core technology: synthetic biology + mRNA
  • Trends in 2021: A round of $52 million financing, cooperation with BeiGene
  • Rating: ⭐⭐⭐⭐

Strand Therapeutics was established in 2017 and the company is located in Cambridge, USA. In 2021, Strand completed a $52 million Series A financing and reached a cooperation with BeiGene on solid tumor mRNA therapy.

Strand is working on the application of synthetic biology to RNA therapeutics, where the synthesized mRNA constructs are constructed by linking self-replicating genes derived from RNA viruses with genetically programmed logic circuits that control the expression (location, timing, and intensity) of therapeutic proteins in patients. circuits) to achieve the specific expression of mRNA in different cell types.

Strand will use mRNA therapy to treat deadly and chronic diseases, initially in solid tumors. Strand plans to initiate human clinical trials of the first drug candidate in 2022.

 

8.Atalanta Therapeutics

  • Core technology: siRNA
  • Trends in 2021: Completion of Series A financing, cooperation with Biogen and Genentech (Roche)
  • Rating: ⭐⭐⭐

Atalanta Therapeutics is located in Boston, USA and was founded in 2018. Professor Craig Mello, one of the founders, is one of the discoverers of the phenomenon of RNA interference. He and Professor Andrew Fire jointly won the 2006 Nobel Prize in Physiology or Medicine.

In January 2021, F-Prime Capital exclusively invested in Atalanta’s A round of financing, and Atalanta also reached a strategic cooperation with Biogen and Genentech, with a total of $110 million in advance payments and financing. The indications for therapy are all neurological diseases.

In addition, Atalanta has licensed the patented branched siRNA technology from the University of Massachusetts Medical School. Preclinical studies have shown that branched siRNAs can perform RNA interference throughout the brain and spinal cord, have potent inhibitory capacity on CNS gene expression, have no detectable toxicity and have minimal off-target effects, and can be applied to a variety of neurological Degenerative diseases.

 

 

9.ProQR Therapeutics

  • Core technology: ASO, RNA editing
  • Key project: sepofarsen (clinical phase II/III)
  • 2021 trends: $1.3 billion partnership with Eli Lilly
  • Rating: ⭐⭐⭐

ProQR Therapeutics was founded in 2012 and went public in 2014. Based in Leiden, The Netherlands, there is a story about the creation of ProQR: The son of founder and CEO Daniel A. de Boer was diagnosed with cystic fibrosis, an incurable genetic disease, shortly after birth , Daniel founded ProQR to help patients with similar diseases.

ProQR has a variety of ASO drugs under development in its pipeline, with indications covering a variety of rare diseases.

 

The September 2021 collaboration between ProQR and Eli Lilly will use RNA editing technology to develop new treatments for genetic diseases of the liver and nervous system.

ProQR’s RNA editing technology is achieved by editing oligonucleotides (EON), which are designed to bind to mRNA at the mutated site, creating a mismatch (non-complementary base pairing), exposing the mutated adenosine (A), and then converting the endogenous adenosine (A).

ADAR enzymes (adenosine deaminase acting on RNA) are recruited to target sites for editing. ADAR will edit adenosine (A) at a specific site to inosine (I), and inosine (I) will be “read” by the translator as guanosine (G), so as to achieve the therapeutic effect of reversing the mutation.

 

10.MiNA Therapeutic

  • Core technology: RNA activation
  • Key Project: MTL-CEBPA (Clinical Phase II)
  • 2021 Trends: Cooperation with Servier and Eli Lilly with a total amount of over 1.5 billion US dollars
  • Rating: ⭐⭐

MiNA Therapeutic is a biotechnology company based in London, UK. Founded in 2008, MiNA Therapeutic is the first company in the world to develop small activating RNA (saRNA) therapy, and its fastest-growing project has entered clinical phase II.

So far, companies that have partnered with MiNA on saRNA therapy include Boehringer Ingelheim, AstraZeneca, Servier and Eli Lilly.

saRNA is a short double-stranded oligonucleotide with a chemical structure similar to siRNA, but the effect is opposite. saRNA works through a completely different RNA activation (RNAa) mechanism, which was first described in humans by Dr. Longcheng Li in 2006. Discovered and named in cells, multiple studies in mammals since then have demonstrated that saRNA can activate multiple genes.

saRNAs exert therapeutic effects by recruiting endogenous transcriptional complexes to target genes, resulting in increased mRNA expression and upregulation of target proteins.

Studies have demonstrated that both naturally expressed and epigenetically silenced target protein levels can be up-regulated following saRNA treatment.

 


Top 10 RNA therapy companies to watch in 2022
The drugs and technologies in the field of RNA therapeutics described herein include various nucleic acid therapeutics in which the drug itself or the drug targets RNA, emerging technologies targeting RNA, and delivery technologies for the above-mentioned therapeutics. The information comes from the PharmaInvest database, NextPharma database and public information. It is for reference only and does not constitute any investment advice. If there are any omissions, please add.

(source:internet, reference only)


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