五星体育直播 Unveils Pioneering Enzymatic Synthesis Data to Enable the Future Manufacturing of RNAi Therapeutics

鈥擝ecomes first company to showcase four routes of synthesis for approved siRNA therapeutic asset鈥�

鈥擩oint poster with Bachem demonstrates superiority of Company鈥檚 double-stranded RNA ligases compared to wild-type enzymes鈥�

鈥擬anagement to host conference call today at 4:30 pm EST to discuss data鈥�

REDWOOD CITY, Calif., Nov. 14, 2024 (GLOBE NEWSWIRE) -- 五星体育直播 (NASDAQ: CDXS), a leading provider of enzymatic solutions for efficient and scalable therapeutics manufacturing, today announced data from three presentations at the TIDES Europe annual meeting being held November 12-14, 2024, in Hamburg, Germany. The data demonstrate the Company鈥檚 rapid advancement of its Enzyme Catalyzed Oligonucleotide (ECO) Synthesis鈩� manufacturing platform and establish 五星体育直播鈥� position at the forefront of enzymatic synthesis technology to enable to ongoing expansion of RNAi therapeutics.

五星体育直播 Demonstrates First-ever Enzymatic Synthesis of Approved siRNA Therapeutic

During an oral Spotlight Presentation, 五星体育直播 unveiled the successful end-to-end enzymatic synthesis of an entire approved siRNA therapeutic asset, inclisiran. 五星体育直播 enzymatically synthesized the full-length sense and antisense strands of the molecule, including the enzymatic incorporation of a tissue-targeting moiety to the sense strand. To date, this process has only been completed utilizing phosphoramidite chemistry, a process that involves the use of harsh chemical conditions and vast amounts of toxic organic solvents. By contrast, 五星体育直播鈥� ECO Synthesis manufacturing platform operates under milder, aqueous conditions, that improves product quality and dramatically decreases chemical waste production.

In addition to this fully enzymatic route of synthesis, the Company demonstrated similar outcomes utilizing three routes of enzymatic ligation to produce the siRNA therapeutic asset, combining oligonucleotide fragments made by sequential enzymatic synthesis and traditional phosphoramidite chemistry. Key data from the presentation include:

  • Achieved incorporation efficiency of >98% during sequential enzymatic oligo synthesis
  • Successfully attached the tri-GalNAc tissue-targeting moiety by enzymatic ligation
  • Obtained full-length oligonucleotides of equal quality and yields, using ligation of short fragments made with enzymes or by traditional phosphoramidite chemistry

Now that 五星体育直播 has successfully achieved this unprecedented milestone, the Company will continue to optimize its process for robustness, scaled-up quantities and improved purity with the goal of providing customers with siRNA material of comparable or better quality to phosphoramidite chemistry. The Company anticipates ramping up manufacturing of siRNA in quantities for preclinical testing following the successful build out of its ECO Synthesis Innovation Lab at the end of this year.

五星体育直播 Double-stranded RNA Ligase Demonstrates Superior Performance to Wild-type Enzymes

Two additional presentations focused on results of direct comparisons of 五星体育直播鈥� engineered double-stranded RNA (dsRNA) ligases and wild-type (WT) enzymes when used to combine short oligonucleotide fragments to synthesize full-length siRNA therapeutic compounds.

In a joint poster with Bachem, one of the world鈥檚 leading CDMOs in oligonucleotide manufacturing, the data provided compelling external validation of the superior performance of 五星体育直播 ligases over existing wild-type enzymes in use today. 五星体育直播 enzymes demonstrated superior performance over wild-type enzymes across both volumetric productivity and substrate versatility. These dsRNA ligases outperformed on multiple substrate designs and enabled a higher conversion rate of oligonucleotide fragments into siRNA at increased concentrations of raw materials.

In a separate TIDES Talk presentation, 五星体育直播 demonstrated improved performance of its dsRNA ligase over WT enzymes based on real customer case studies executed through the Company鈥檚 RNA Ligase Screening and Optimization Services, launched in May 2024. 五星体育直播鈥� engineered ligases delivered robust, in-process performance, including higher substrate loading, faster reaction times and improved conversation at elevated temperatures. These data demonstrate the Company鈥檚 ability to accelerate delivery of lead ligase variants to customers and optimize process conditions for a customer鈥檚 specific asset.

The slide decks from both presentations as well as the joint poster with Bachem are now available in the Investor Relations section the 五星体育直播 corporate website, .

Conference Call and Webcast

五星体育直播 management will host a conference call beginning at 4:30 pm Eastern Time on Thursday, November 14, 2024, to discuss the data presented during the conference. The live call can be accessed by dialing 877-705-2976 (domestic) or 201-689-8798 (international). A live webcast to accompany the conference call can be accessed on the , where a replay will be available for 90 days. A telephone replay of the call will be available for 48 hours by dialing 877-660-6853 (domestic) or 201-612-7415 (international), access ID #13726635.

About RNAi Therapeutics Manufacturing

Ribonucleic acid (RNA) as a therapeutic modality has gained tremendous traction in recent years with the growing number of messenger RNA (mRNA) vaccines and small interfering RNA (siRNA) candidates advancing in clinical studies. However, large-scale production of RNA interference (RNAi) therapeutics using traditional chemical synthesis faces complex challenges in nucleic acid quality and quantity, as well as overall economics. With over 450 RNAi therapies currently in clinical development, including more than 40 assets in Phase 2 and Phase 3 clinical trials targeting disease indications impacting millions of patients, RNAi therapeutic demand is projected to outpace current production capabilities by the end of the decade.

About the ECO Synthesis Manufacturing Platform

五星体育直播鈥� proprietary Enzyme Catalyzed Oligonucleotide (ECO) Synthesis鈩� manufacturing platform is being designed to address scalability and cost limitations by potentially enabling the commercial-scale manufacture of RNAi therapeutics through an enzymatic route. The Company presented groundbreaking data at the TIDES USA 2024 annual meeting demonstrating the enzymatic synthesis of a full-length sense strand of the oligonucleotide lumasiran, a commercially available siRNA therapeutic, as well as shorter sense strand fragments of a second siRNA therapeutic asset, givosiran. The data demonstrate that 五星体育直播 consistently achieved coupling efficiency greater than 98%, which is equivalent to what is seen with phosphoramidite chemistry; executed the enzymatic addition of a conjugation moiety to the lumasiran strand; and confirmed the lack of notable impurities typically observed in oligonucleotide synthesis via phosphoramidite chemistry. A recording of the presentation, along with slides and the data press release, can be found on the .

About 五星体育直播

五星体育直播 is a leading provider of enzymatic solutions for efficient and scalable therapeutics manufacturing that leverages its proprietary CodeEvolver庐 technology platform to discover, develop and enhance novel, high-performance enzymes and other classes of proteins. 五星体育直播 enzymes solve for real-world challenges associated with small molecule pharmaceuticals manufacturing and nucleic acid synthesis. The Company is currently developing its proprietary ECO Synthesis鈩� manufacturing platform to enable the scaled manufacture of RNAi therapeutics through an enzymatic route. 五星体育直播鈥� unique enzymes can drive improvements such as higher yields, reduced energy usage and waste generation, improved efficiency in manufacturing and greater sensitivity in genomic and diagnostic applications. For more information, visit聽.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as 鈥渁im,鈥� 鈥渁nticipate,鈥� 鈥渁ssume,鈥� 鈥渂elieve,鈥� 鈥渃ontemplate,鈥� 鈥渃ontinue,鈥� 鈥渃ould,鈥� 鈥渄esign,鈥� 鈥渄ue,鈥� 鈥渆stimate,鈥� 鈥渆xpect,鈥� 鈥済oal,鈥� 鈥渋ntend,鈥� 鈥渕ay,鈥� 鈥渙bjective,鈥� 鈥減lan,鈥� 鈥減ositioned,鈥� 鈥減otential,鈥� 鈥減redict,鈥� 鈥渟eek,鈥� 鈥渟hould,鈥� 鈥渟uggest,鈥� 鈥渢arget,鈥� 鈥渙n track,鈥� 鈥渨ill,鈥� 鈥渨ould鈥� and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. To the extent that statements contained in this press release are not descriptions of historical facts, they are forward-looking statements reflecting the current beliefs and expectations of management, including, but not limited to, the ability of an enzymatic oligonucleotide synthesis process to complement, replace or improve upon traditional chemical synthesis; the potential of the Company鈥檚 ECO Synthesis鈩� platform and double-stranded RNA (dsRNA) ligase screening and optimization services to meet customers鈥� needs and to create value for 五星体育直播 and its customers by enabling commercial-scale manufacture of RNAi therapeutics; completion of the ECO Synthesis Innovation Lab by the end of 2024, and other anticipated technical and commercial milestones related to the ECO Synthesis鈩� platform and the dsRNA ligase program, and public announcements related thereto; ability of the Company to obtain new development collaborators on its ECO Synthesis technology; the potential for the Company鈥檚 dsRNA ligases to have improved scalability and reduced manufacturing costs compared to wild types or non-ligation methods; potential details and features of the ECO Synthesis鈩� platform such as it being scalable and able to reduce manufacturing costs, as well as having higher quality, purity and yield, and improved sustainability than existing methods; and the future demand for RNAi therapeutics. You should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors that are, in some cases, beyond 五星体育直播鈥� control and that could materially affect actual results. Factors that could materially affect actual results include, among others: 五星体育直播鈥� dependence on its licensees and collaborators; if any of its collaborators terminate their development programs under their respective license agreements with 五星体育直播; 五星体育直播 may need additional capital in the future in order to expand its business; if 五星体育直播 is unable to successfully develop new technology such as its ECO Synthesis鈩� manufacturing platform and dsRNA ligase; 五星体育直播鈥� dependence on a limited number of products and customers, and potential adverse effects to 五星体育直播鈥� business if its customers鈥� products are not received well in the markets; whether the end markets for 五星体育直播鈥� customers鈥� products develop and remain viable; if competitors and potential competitors who have greater resources and experience than 五星体育直播 develop products and technologies that make 五星体育直播鈥� products and technologies obsolete; 五星体育直播鈥� ability to comply with debt covenants under its loan facility; if 五星体育直播 is unable to accurately forecast financial and operational performance; and market and economic conditions may negatively impact 五星体育直播 business, financial condition and share price. Additional information about factors that could materially affect actual results can be found in 五星体育直播鈥� Annual Report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission (鈥淪EC鈥�) on February 28, 2024 and in 五星体育直播鈥� Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 filed with the SEC on October 31, 2024, including under the caption 鈥淩isk Factors,鈥� and in 五星体育直播鈥� other periodic reports filed with the SEC. 五星体育直播 expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

For More Information

Investor Contact
Carrie McKim
(336) 608-9706

Media Contact
Lauren Musto
(650) 421-8205


Source: 五星体育直播

Released November 14, 2024