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Aptamers in Personalized Medicine: Tailoring Treatment for Better Patient Outcom

<br>The aptamers market is estimated to be worth $451 million in 2024, driven by launch of Izervay and is expected to grow at compounded annual growth rate (CAGR) of 36% during the forecast period 2024-2035. Aptamer technology and services market is estimated to be $58 million in 2022, growing at double-digit CAGR consistent with the market trends in aptamer therapeutics. Post the approval of the first aptamer-based therapy, Macugenu00ae, by the US Food and Drug Administration (USFDA) in December 2004, there has been a significant increase in the R&D initiatives focused on evaluating the potential of

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Aptamers in Personalized Medicine: Tailoring Treatment for Better Patient Outcom

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  1. Tracking the healthcare innovation Free Insights Aptamers: Therapeutics, Technologies and Services Market, 2022-2035 Distribution by [A] Application Area (Therapeutics, Research and Diagnostics), [B] Type of Aptamer (DNA, RNA and Peptide), [C] Target Indications (Brain Cancer, Geographic Atrophy, Hemophilia A, Macular Degeneration, Pancreatic Cancer, Von Willebrand Diseases), [D] Route of Administration (Intravitreal, Intravenous, Subcutaneous), [E] Scale of Operation (Small Scale, Medium Scale and Large Scale), [F] Focus Area (Product Development, Product Commercialization, Technology Integration, Technology Utilization and Others), [G] Company Size (Very Small, Small, Mid-sized, Large and Very Large) and [H] Key Geographical Regions (North America, Europe, Asia-Pacific and Rest of the World) C O N F I D E N T I A L | www.rootsanalysis.com

  2. Context Upon realizing the potential of aptamers over antibodies, industry stakeholders have initiated several R&D efforts focused on exploiting the use of aptamers for various purposes such as detection, diagnosis, treatment and research 40+ Drug Candidates >450 50+ Service Providers Given the various advantages offered by this novel drug class, the demand for aptamer-based therapeutics is expected to witness a notable surge, presenting lucrative opportunities for contract services providers in the coming decade Patents Overview of Aptamers Market Currently being evaluated across various stages of development Filed by various industry and non-industry stakeholders since 2016 Have required capabilities to cater to the needs of drug developers Non-immunogenic Targets Targets Small Molecules Stability and Storage Aptamers have recently emerged as a promising target for the development of various therapies; several candidates in the development pipeline have demonstrated considerable benefits over conventional antibody-based treatment methods Advantages of Aptamers Over Antibodies Enables targeting molecules that are ten times smaller than those targeted by antibodies Aptamer synthesis does not involve living cells, allowing for their use against toxic compounds These molecules can be stored easily, as they are resistant to denaturation at high temperatures USD Billion 50+ 1.3+ >25% Aptamer Technologies Driven by encouraging clinical trial results and the growing demand for safe and cost-effective treatments, along with development of novel technologies, the aptamers market is anticipated to witness accelerated growth, over the next decade CAGR Current Industry Landscape and Upcoming Trends Currently being developed for a range of application areas Has been invested to finance R&D efforts in this domain, over the last few years Indicative of the high growth opportunity in the aptamer-based therapeutics market

  3. Example Highlights Presently, more than 40 aptamer-based drug candidates are being investigated across different phases of development for the treatment of a myriad of disease indications Full list of 40+ aptamer-based therapies, along with information on several parameters is available in an excel format upon request List of Aptamer-based Therapeutic Candidates1 Developer Landscape: Distribution by Company Size and Location of Headquarters2 56% North America 31% Europe Note 1: Macugen was approved by the United States Food and Drug Administration in December 2004 Note 2: Players were categorized based on company size (in terms of number of employees) as : Very Small: 2-10, Small:11-50, Mid-sized: 51-200, Large: 201-5,000, Very Large: 5,000+ 13% Asia-Pacific

  4. Example Highlights Most of the aptamer drugs are being developed as RNA-based therapies; 46% of the aptamer-based therapies are in pre-clinical stage and are being developed for various therapeutic areas • Market Landscape: Aptamer-based Therapy Candidates • Distribution by Type of Aptamer1 • Market Landscape: Aptamer-based Therapy Candidates • Distribution by Phase of Development2 • Market Landscape: Aptamer-based Therapy Candidates • Distribution by Therapeutic Area3,4 RNA Aptamers DNA Aptamers 61% 24% L-RNA Aptamers Peptide Aptamers 11% 4% Note 1: Therapies for which information on type of aptamer was available have been included in this representation Note 2: Therapies for which information on phase of development was available have been included in this representation Note 3: Therapies for which information on therapeutic area was available have been included in this representation • Note 4: Therapies targeting more than one therapeutic area have been counted multiple times in this representation

  5. Example Highlights Foreseeing lucrative returns, many public and private investors have made investments worth USD 1.3 billion since 2016; the funding activity is well distributed across geographical regions • Fundings and Investments • Cumulative Distribution of Instances • Funding and Investments • Distribution by Amount Invested and ToF2 • Funding and Investments • Distribution by Amount Invested and LoR USD Million 1.3+ USD Billion Invested Others Seed Financing Debt Financing Other Equity Private Placement Awards / Grants Secondary Offering Venture Capital USD 442 Million Invested in 2021 41% 28% Funding instances in Europe 2016 2017 2018 2019 2020 2021 31% Funding instances in North America Funding instances in Asia-Pacific XX Legend XX XX • Example Investors 60.5 XX XX XX 8.4 XX XX 43 60+ Investors XX XX XX XX 54.7 XX • Abbreviations: FI: Funding Instances; ToF: Type of Funding; LoR: Location of Recipient XX 5.2 XX XX XX 7.3

  6. Example Highlights A considerable increase in partnership activity has been witnessed over the past few years, indicating a steady rise in the interest of stakeholders • Partnerships and Collaborations • Year-wise Trend, 2016-20211,2 • Partnerships and Collaborations • Distribution by Type of Partnerships2 • Partnerships and Collaborations • Country-wise Distribution2 Type of Partnerships in 2021: 36% 23% 29% Research and Development Agreements Product Development Agreements Service Agreements CAGR: 20% (2016-2021) Example Partnership: October 2021 Focus Area R & D Agreement As per the terms of the agreement, both firms agreed to combine their technologies for the discovery and development of novel aptamer therapeutics s s s s • Note 1: 98 instances for which information on year of partnership was available have been included in this representation • Note 2: Information in this report (specifically this table) has been identified from publicly available sources on a ‘best-effort’ basis. However, we realize that some of the data points may not be publicly available, and as such, may have been overlooked in our analysis. If you would like to notify us of these gaps, please send an email to support@rootsanalysis.com. s s s s s

  7. Example Highlights Start-ups are spearheading the innovation in this domain; our proprietary benchmarking analysis, based on a variety of parameters, highlights the leading players Start-up Health Indexing: Technology Providers Stat-up Benchmarking1 Start-up Health Indexing: Technology Providers Wind Rose Chart: Top 9 Players Content Illustrative Company P1 P2 • P3 • P4 • P5 • P6 • Total Score (details available upon request) Very Small Very Small Small Small Mid-sized Mid-sized Developer 1 Developer 3 Developer 5 Developer 7 Developer 8 Developer 10 Distribution by Company Size and Location of Headquarters 2 Developer 12 Developer 13 Developer 14 Developer 15 Developer 16 Note 1: Parameters considered for this analysis includes P1: Number of technologies, P2: Type of Aptamer, P3: Status of Development, P4: Ara of Application, P5: Types of Bioanalytical Technology Used, P6: Technology Application Area Note 2: Start-ups for which information on company size was available have been included in this representation ~45.5% Aptamer-based technology developers are located in North America

  8. Example Highlights 400+ academic grants have been awarded to organizations over the past five years to support the ongoing efforts related to research and innovation in the field of aptamers Academic Grant Analysis Cumulative Year-wise Trend1,2 Academic Grant Analysis Distribution by Activity Codes1,2,3 Academic Grant Analysis Word Cloud: NIH Spending Category5 USD Million Cumulative Distribution of Grant Amount 20 47 75 107 137 181 Academic Grants: Distribution by Support Period4 USD 44 Million 24.3% granted in the year 2021 Program Project / Center Grants (P Series) Note 1: 424 instances have been included in this representation • Note 2: The data has been collected through publicly available sources • Note 3: Others category includes activity codes for which specific information was not available • Note 4: Support period refers to the duration (calculated as the difference between start year and end year) for which a particular grant was awarded to a research institute / academic research group • Note 5: The representation highlights the keywords that appeared most frequently in the grants captured during our analysis

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