麻豆原创

RNA-Targeted Small Molecules: Emerging Discoveries are Catching the Eye of Investors

RNA-Targeted Small Molecules: Emerging Discoveries are Catching the Eye of Investors
One of the popular states of New England, Massachusetts has become the cradle of innovation in small molecule therapies.Companies such as Accent Therapeutics, Arrakis Pharmaceuticals, H3 Biomedicine, and Twentyeight-Seven Therapeutics have attracted substantial investments in recent years.

Leading Therapeutic Companies in Field of RNA-Targeted Small Molecules

  • Accent Therapeutics
  • Anima Biotech Inc.
  • Arrakis Pharmaceuticals
  • Epics Therapeutics
  • Expansion Therapeutics
  • Gotham Therapeutics
  • H3 Biomedicine
  • Ribometrix
  • Skyhawk Therapeutics
  • STORM Therapeutics
  • Twentyeight-Seven Therapeutics

Leading Therapeutic Companies in Field of RNA-Targeted Small Molecules

Since 2017, investors and Big Pharma companies have poured nearly USD 1 billion to finance emerging companies with enabling technologies for RNA biology and for discovering small molecules against RNA as a drug target. This huge amount of money highlights the tremendous interest from investors and major pharmaceutical companies and the opportunities they recognize in these new approaches to target RNA with small molecules.
Nearly all small molecule-based drugs in clinical use target proteins. It is estimated that about 20,000 human proteins are expressed by the human genome and 10-15% are thought to be disease-related. However, many of them are considered undruggable for various reasons, e.g. because they lack a distinctive motif for small molecule binding.
RNA as a target holds the promise of dramatically increasing the pool of druggable targets as compared with conventional protein targets. RNA as a therapeutic drug has validated this concept听but has limitations such as delivery issues and toxicity. Thus, small molecules targeting RNA is an attractive concept for the pharmaceutical industry.
RNA-Targeted Small Molecules 麻豆原创 Research Report

The RNA-Targeted Small Molecules 麻豆原创 report describes and analyzes from an industry perspective with a view on emerging technology companies regarding their founding background, financial history, technological capabilities, target and indication selection, and most advanced lead compounds.

This report is very useful for decision-makers, venture capital, private equity and investment managers in Big Pharma companies, R&D Portfolio, Technology, and Strategy Management and clinical specialists.

Four approaches to target RNA with small molecules are separately examined in the report:听
  • mRNA translation regulation;
  • RNA splicing modification
  • Direct RNA targeting; and
  • Epitranscriptomics, i.e. indirectly targeting RNA via the proteins it is interacting with.
Albeit different, the four approaches are complementary, but the analysis shows differences in the maturity and preferences of pharma partners and investors.听Big Pharma also has in-house R&D activities to discover small molecules targeting RNA complemented with technology collaborations and support of emerging technologies via their venture capital. The report describes the stakes Big Pharma holds in the field and the level of participation.

Recent Investment in RNA-Targeted Small Molecules Therapeutics

  • In September 2019, Ribometrix has announced USD 7.8 million in additional funding from existing investors, the Dementia Discovery Fund and Illumina Ventures. The new funding will strengthen the ongoing development of the Ribometrix discovery platform, which uses sophisticated structural analysis and proprietary assays to identify ligands that bind to highly structured pockets in therapeutically compelling RNAs to modulate protein expression.
  • In May 2019, STORM Therapeutics announced that it has raised an additional 拢14 million bringing the total Series A financing to 拢30 million (USD 38.43 million). The funding will enable STORM to advance its current pipeline further in preclinical development and accelerate its programmes towards the clinic, supporting STORM's growth as the leading drug discovery company working on RNA modulating enzymes.
  • On April 2019, Arrakis Therapeutics announced USD 75 million Series B financing to Advance a New Class of Small-Molecule Medicines Targeting RNA
  • In December 2018, EPICS Therapeutics SA announced that it will raise Euro 14.2 million (USD 16.1 million) to fund its discovery and development of innovative small molecules anti-cancer drugs.
  • In October 2018, Gotham Therapeutics was formed with a USD 54 million Series A financing co-led by founding investor Versant Ventures, Forbion and S.R. One. Gotham Therapeutics will be developing a novel drug class targeting RNA-modifying proteins.
  • In July 2018, Anima Biotech entered an agreement with Lilly for the discovery and development of translation inhibitors for several target proteins by using Anima's Translation Control Therapeutics platform. Under the terms of the agreement, Anima will receive USD 30 million in upfront payments and USD 14 million in research funding.
  • In May 2018, Accent Therapeutics announced USD 40 million in Series A capital to establish a discovery platform and pipeline of therapeutic candidates targeting RMPs.
  • On January 2018 Expansion Therapeutics, Inc. closed of a USD 55.3 million Series A funding co-led by 5AM Ventures, Kleiner Perkins, Novartis Venture Fund, and Sanofi Ventures with participation from RA Capital Management and Alexandria Venture Investments. The funding will strengthen the Expansion鈥檚 portfolio of small molecule drugs targeting key human disease-driving RNAs.

Disclaimer: The article has been compiled based on the data provided in " RNA-Targeted Small Molecules 2019" report and press release issued by respective companies.

Related Biotech Industry Articles

1.听Amgen and Roche to Lead Global Bispecific Antibodies 麻豆原创

2.听Top 10 Oncology Pharma Companies in the World

3.听Top 5 Biotech Companies to Look out for Adrenoleukodystrophy (ALD)

Contact Us for Custom 麻豆原创 Research Solutions

麻豆原创ResearchReports.com Newsletter Subscription

About The Author

sudeep's picture
Sudeep Chakravarty

Feature your company here

Clients Who Trust Us

麻豆原创 Research Reports Inc. Customers

Need tailor made market research solution? We can help you with that too.

About Us

At 麻豆原创 we aim to make it easier for decision makers to find relevant information and locate right market research reports which can save their time and assist in what they do best, i.e. take time-critical decisions.

We work with our associate Global market research firms who are known leaders in their respective domains to obtain right market research solution for our customer鈥檚 needs, be it custom research or syndicated research reports.

Contact Us

麻豆原创 Research Reports, Inc
16192 Coastal Hwy
Lewes
, DE 19958, USA

USA: +1-302-703-9904

India: +91-8762746600

marketresearchreports

info@marketresearchreports.com

User login

Stay Connected