Exicure, Inc. Reports First Quarter 2022 Financial Results and Corporate Progress

On May 16, 2022 Exicure, Inc. (NASDAQ: XCUR), an early-stage biotechnology company focused on the development of next generation nucleic acid therapies targeting RNA to address both genetic and non-genetic neurological disorders and hair loss disorders, reported financial results for the quarter ended March 31, 2022 and provided an update on its business strategy and corporate progress (Press release, Exicure, MAY 16, 2022, View Source [SID1234614704]).

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"Exicure is off to a promising start in 2022 as we have made progress with our preclinical SCN9A program for the treatment of pain with several potential therapeutic candidates identified and we are conducting initial in vivo animal studies to support candidate selection in 2023," commented Matthias Schroff, Ph.D., Chief Executive Officer of Exicure. "We continue to advance our partnered programs with Ipsen and AbbVie, and the recently announced private placement investment led by CBI USA, Inc. ("CBI USA") is expected to provide us with additional resources as we continue our mission to pursue treatments for patients with unmet medical needs," concluded Dr. Schroff.

Corporate Progress

Recent highlights include:

On May 10, 2022, Exicure announced a $5.0 million raise in a private placement transaction priced at market premium
Agreed to sell an aggregate of 26,021,011 shares of the Company’s common stock to certain accredited investors in a private placement in public equity ("PIPE") financing at a purchase price of $0.1937 per share, representing an approximately 45% premium to the 10-day volume weighted-average share price from May 9, 2022.
New investor CBI USA led the transaction; existing investor, Abingworth LLP, also participated.
Transaction is expected to close on or about May 19, 2022, subject to the satisfaction of customary closing conditions.
In connection with the PIPE, CBI USA received the right to nominate a member to the Company’s board of directors (the "Board"), effective as of the closing date. CBI USA will also have the right to designate one individual to attend all meetings of the Board in a nonvoting observer capacity.
Net proceeds from the transaction expected to support the Company’s advancement of its preclinical program, including the development of its SCN9A product candidate, as well as other working capital and general corporate purposes.
Corporate highlights for the first quarter of 2022 include:

Repaid in full all outstanding indebtedness and other obligations under the Company’s credit facility with MidCap, effective March 15, 2022.
Continued to advance the Company’s SCN9A preclinical discovery program. Exicure anticipates results from initial in vivo animal studies by year-end 2022, with the goal of therapeutic candidate selection in the second half of 2023.
Progressed work with partnered programs towards potential pre-clinical milestones in 2023.
Actively pursuing out-license opportunities for the Company’s clinical asset, cavrotolimod.
Continuing to pursue near-term partnering opportunities for pain and other neuroscience programs.
First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and short-term investments were $27.6 million as of March 31, 2022, as compared to $48.3 million as of December 31, 2021. The Company expects that its cash and cash equivalents, together with the expected $5.0 million gross proceeds from the PIPE transaction in May 2022, will enable it to fund its current operations into the first quarter of 2023.

Revenue: Revenue was $2.6 million for the quarter ended March 31, 2022, reflecting an increase of $1.6 million from revenue of $1.0 million for the quarter ended March 31, 2021. The increase in revenue of $1.6 million is mostly due to the recognition of non-cash revenue of $2.1 million associated with the Company’s collaboration with Ipsen Biopharm Limited, partially offset by a decrease in revenue of $0.5 million associated with the Company’s collaboration with AbbVie Inc.

Research and Development (R&D) Expense: Research and development expenses were $7.1 million for the quarter ended March 31, 2022, as compared to $10.3 million for the quarter ended March 31, 2021. The decrease in R&D expense for the three months ended March 31, 2022 of $3.1 million reflects a reduction in employee headcount and fewer discovery, preclinical, and clinical program activities resulting from the restructuring activities that the Company announced in December 2021.

General and Administrative (G&A) Expense: General and administrative expenses were $3.2 million for the quarter ended March 31, 2022, as compared to $2.9 million for the quarter ended March 31, 2021. The increase in G&A expense of $0.3 million for the three months ended March 31, 2022 was mostly due to higher legal costs and retention award expense for current employees, partially offset by a decrease in recruiting costs, investor relations costs, and stock-based compensation.

Net Loss: The Company had a net loss of $8.3 million for the quarter ended March 31, 2022, as compared to a net loss of $12.5 million for the quarter ended March 31, 2021. The decrease in net loss was primarily driven by lower R&D expense and higher non-cash revenue during the period.

Going Concern: Given the Company’s current cash position, operating plans and forecasted negative cash flows from operating activities over the next twelve months, management believes there is substantial doubt regarding the Company’s ability to continue as a going concern within one year after the date that its unaudited condensed consolidated financial statements for the quarter ended March 31, 2022 are issued. The Company will require substantial additional financing to address the Company’s working capital and other financing needs to pursue its business strategy.

Centessa Pharmaceuticals Reports First Quarter 2022 Financial Results and Provides Business Updates

On May 16, 2022 Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company with a Research & Development ("R&D") innovation engine that aims to discover, develop and ultimately deliver impactful medicines to patients, reported financial results for the first quarter ended March 31, 2022 and provided business updates (Press release, Centessa Pharmaceuticals, MAY 16, 2022, View Source [SID1234614702]).

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"We continue to execute against our focused strategy and advance our pipeline of innovative, high impact rare disease and immuno-oncology assets that we believe will position Centessa for multiple value-creating milestones through our extended cash runway. Our model is focused on judicious capital and resource allocation decisions which, combined with our strong balance sheet, provide us with significant optionality to meet our ‘4×24’ goal of four registrational programs in 2024," said Saurabh Saha, MD, PhD, Chief Executive Officer of Centessa. "Our most advanced program, lixivaptan for ADPKD, continues to progress in the ongoing registrational ACTION Study. In addition, ZF874 for AATD is on track for data later this year from the ongoing Phase 1 study, we continue to anticipate SerpinPC in Hemophilia to enter registrational trials this year, and we recently commenced dosing in the Phase 1 study of CBS001, an anti-LIGHT monoclonal antibody. In the near term, we are excited to share initial preclinical data for LB101, our PD-L1xCD47 LockBody at ASCO (Free ASCO Whitepaper) 2022 in June."

First Quarter 2022 Highlights and Recent Business Updates
Clinical Development Programs:
•SerpinPC: Based on the FDA feedback received in the first quarter of 2022, the Company is proceeding with a streamlined, integrated registrational development plan initially for Hemophilia B (with and without inhibitors).
•Lixivaptan: Commenced dosing in the pivotal Phase 3 clinical trial ("ACTION Study") evaluating lixivaptan as a potential treatment for ADPKD. In addition, a key US patent was issued on

February 8, 2022, which covers the use of lixivaptan for the treatment of ADPKD. The patent term expires June 8, 2038, before considering possible patent term extensions or adjustments.
•CBS001 in inflammatory / fibrotic diseases: Commenced dosing of healthy volunteers in a Phase 1 study of CBS001, a high affinity anti-LIGHT monoclonal antibody.

Business Highlights:
•During the first quarter of 2022, the Company named Antoine Yver, MD, MSc, Executive Vice President and Chairman of Development; Javad Shahidi, MD, MSc, Chief Medical Officer; and Josh Hamermesh, MBA, Senior Vice President, Business Development.
•In March 2022, the Company announced ‘4×24’, its corporate goal to have four registrational programs in 2024.
•In March 2022, the Company announced plans to discontinue the small molecule EGFR inhibitor discovery programs (PearlRiver Bio) and the dual-STAT3/5 degrader program (Janpix Limited) and is now winding down operations at both subsidiaries. The Company also continues to evaluate strategic options, including potential divestment, for imgatuzumab (Pega-One).
•In February 2022, dosing of the first patient with lixivaptan in the Phase 3 ACTION Study triggered settlement of the contingent value rights ("CVRs") originally issued to the former shareholders and option holders of Palladio Biosciences in connection with its acquisition by Centessa in January 2021.

Anticipated Upcoming Program Milestones
•LB101 in Solid Tumors: Preclinical data for LB101, a PD-L1xCD47 LockBody, will be presented in a poster at ASCO (Free ASCO Whitepaper) 2022 on June 5, 2022. An IND for LB101 is planned for late 2022.
•ZF874 in Alpha-1 Antitrypsin Deficiency (AATD): The Company expects to share data from the ongoing Phase 1 study in the second half of 2022. This interim data will include multiple dose cohorts with PiMZ and PiZZ subjects.
•SerpinPC in Hemophilia: In the second half of 2022, the Company expects to initiate a streamlined clinical program to support registration for SerpinPC for the treatment of Hemophilia B (with and without inhibitors). Additionally, the Company expects to report data from the Phase 2a open label extension study in the fourth quarter of 2022 for the 48-week flat dose portion and the 24-week high dose portion (1.2mg/kg every two weeks). This is anticipated to provide two years of safety and efficacy data across multiple dose levels.
•CBS004 in Autoimmune Diseases: IND is planned for late 2022.

First Quarter 2022 Financial Results
•Cash and Cash Equivalents: $544.5 million as of March 31, 2022 which the Company expects will fund operations to mid-2024, without drawing on the remaining available tranches under the Oberland credit facility.

•Research & Development Expenses: $36.9 million for the quarter ended March 31, 2022 compared to $10.1 million for the period from January 30, 2021 through March 31, 2021.
•General & Administrative Expenses: $14.4 million for the quarter ended March 31, 2022 compared to $5.6 million the period from January 30, 2021 through March 31, 2021.
•Net Loss Attributable to Ordinary Shareholders: $54.5 million for the quarter ended March 31, 2022 compared to $238.7 million for the period from January 30, 2021 through March 31, 2021 (which includes a special non-cash $220.5 million charge for acquired in-process R&D associated with the Centessa subsidiary acquisitions).

Adamis Pharmaceuticals Reports First Quarter 2022 Financial Results and Provides Corporate Update

On May 16, 2022 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP), a biopharmaceutical company developing and commercializing specialty products for allergy, opioid overdose, respiratory and inflammatory disease, reported financial results for the quarter ending March 31, 2022 (Press release, Adamis Pharmaceuticals, MAY 16, 2022, View Source [SID1234614699]).

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Product and Pipeline Updates and Other Corporate Developments

ZIMHI

·According to the Centers for Disease Control and Prevention (CDC), drug overdoses resulted in over 108,000 deaths in the U.S. over the most recent 12 months of data. Two thirds of these involved fentanyl.

·Our U.S. commercial partner, US WorldMeds, commercially launched our high dose naloxone product, ZIMHI, at the end of March.

·We are encouraged by the early acceptance of ZIMHI in the market.

SYMJEPI

·In March, Adamis announced a voluntary recall of certain lots of SYMJEPI.

·Manufacturing of SYMJEPI has been on hold pending the results of an investigation to determine the root cause. The Company believes the investigation is nearing completion, that a root cause relating to a particular batch of syringe needles has been identified, and that corrective and preventive actions have been and will be taken.

·We anticipate a resolution and resumption of manufacturing after those issues are satisfactorily addressed, which we believe will occur during the second quarter.

TEMPOL

·The Company’s Phase 2/3 clinical trial of Tempol as a treatment for COVID-19 is continuing.

·In March, the Data Safety Monitoring Board (DSMB) overseeing the Tempol trial met to evaluate the clinical and safety data from the first planned interim analysis and, following their evaluation, recommended the study continue without modification.

·The DSMB plans to meet again, anticipated to be at the end of May or in June, to review interim data analysis for the first 124 patients.

·In addition to the work in COVID, the Company continues to explore additional indications for the use of Tempol including, but not limited to, asthma and long COVID.

Financial Results

Revenues for the quarters ending March 31, 2022 and 2021 were approximately $1.2 million and $1.4 million, respectively. Revenues for the quarter ended March 31, 2022 consisted mainly of approximately $1.1 million of sales of ZIMHI to our commercial partner US WorldMeds in anticipation of the commercial launch of ZIMHI announced at the end of March. Due to the SYMJEPI manufacturing hold and the voluntary recall of certain lots, no revenues relating to SYMJEPI were reported for the first quarter of 2022.

Selling, general and administrative expenses for the quarters ending March 31, 2022 and 2021 were approximately $3.4 million and $3.5 million, respectively. SG&A expenses in the first quarter of 2022 reflected a decrease in legal and compensation expenses, offset by an increase in accounting and finance related expenses.

Research and development expenses were approximately $4.2 million and $2.2 million for the first quarter of 2022 and 2021, respectively. The increase was primarily due to expenses relating to the ongoing clinical trial for our Tempol product candidate.

Net loss from discontinued operations for the three months ended March 31, 2022 and 2021 was approximately $165,000 and approximately $1.5 million, respectively. The decrease in loss was primarily attributable to the winding down and cessation of US Compounding’s operations.

Cash and cash equivalents at March 31, 2022, totaled approximately $17.8 million. For this year, we expect to receive additional proceeds resulting from amounts payable to us pursuant to our sale of certain USC assets to Fagron and from the disposition of the remaining USC assets which includes the land, the building, the machinery and the equipment.

Conference Call

Adamis will host a conference call and live webcast today, May 16, 2022, at 2 p.m. PT (5 p.m. ET) to discuss its financial and operating results for the first quarter 2022, as well as provide an update on business developments and activities.

A live audio webcast of the conference call will also be available via this link. If you are unable to participate in the live call, a replay will be available shortly after the live event. To listen to the replay please visit the events page of the Adamis investor relations section of the company website at View Source

Faron Announces US Food and Drug Administration and Finnish Medicines Agency Approval to Initiate Phase I/II Bexmarilimab Combination Study in Hematologic Malignancies

On May 16, 2022 Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical stage biopharmaceutical company focused on building the future of immunotherapy by harnessing the power of the immune system to tackle cancer and inflammation, reported that both the U.S. Food and Drug Administration (FDA) and Finnish Medicines Agency (FIMEA) have cleared Faron’s Investigational New Drug (IND) application to begin the Company sponsored BEXMAB study (Press release, Faron Pharmaceuticals, MAY 16, 2022, View Source [SID1234614687]). BEXMAB is a novel Phase I/II study to assess safety, tolerability and preliminary efficacy of bexmarilimab, Faron’s wholly-owned investigational precision cancer immunotherapy, in combination with standard of care (SoC) therapy in patients with relapsed acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelomonocytic leukemia (CML). This marks the first time bexmarilimab will be assessed as part of a clinical study in hematologic malignancies.

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"We are pleased that our IND application was cleared to proceed, and we can further explore the strong scientific rationale for combining bexmarilimab and azacitidine." said Marie-Louise Fjällskog, M.D., Ph.D., Chief Medical Officer of Faron. "Research has shown a clear survival benefit among certain blood cancer patients with low Clever-1 expression, a receptor known to be expressed on immunosuppressive macrophages in the tumor microenvironment. By adding bexmarilimab to standard of care we expect to downregulate Clever-1 expression, thereby increasing antigen presentation and allowing the immune system to better identify and kill cancer cells."

The primary objective of the BEXMAB study is to determine the safety and tolerability of bexmarilimab in combination with SoC treatment and to identify the recommended Phase 2 dose. Secondary objectives include characterizing the pharmacokinetic profile of bexmarilimab in combination with SoC treatment (azacitidine) and to assess the immunogenicity of bexmarilimab. Based on initial safety data, there is potential for Phase II expansion and to include a first line triplet therapy of bexmarilimab, azacitidine and venetoclax in newly diagnosed AML patients who are not able to tolerate chemotherapy. Patient recruitment is expected to begin in the coming weeks.

"We know from pre-clinical research, some of which was presented recently at EHA (Free EHA Whitepaper), that certain blood cancer cells, especially with myeloid background, carry significant amounts of cell surface Clever-1," said Dr. Markku Jalkanen, Chief Executive Officer of Faron. "This corresponds with the presence of considerable amounts of soluble Clever-1, which limits T cell activation leading to a possible systemic loss of immune capacity. Directly targeting Clever-1 could ignite the immune system, limit the replication capacity of cancer cells, and allow current chemotherapy treatments to be more effective."

In addition to the BEXMAB study focused on hematologic malignancies, Faron is also investigating bexmarilimab in solid tumors. The ongoing Phase I/II MATINS clinical trial is assessing bexmarilimab as a potential monotherapy in late-stage, heavily pre-treated patients across multiple tumor types. Additionally, the Company expects to initiate a trial assessing the safety and tolerability of bexmarilimab in combination with an approved anti-PD-1 molecule in multiple solid tumors later this year.

About Bexmarilimab

Bexmarilimab is Faron’s wholly-owned, investigative precision immunotherapy with the potential to provide permanent immune stimulation for difficult-to-treat cancers through targeting myeloid cell function. A novel anti-Clever-1 humanised antibody, bexmarilimab targets Clever-1 positive (Common Lymphatic Endothelial and Vascular Endothelial Receptor 1) tumour associated macrophages (TAMs) in the tumour microenvironment, converting these highly immunosuppressive M2 macrophages to immune stimulating M1 macrophages. In mouse models, bexmarilimab has successfully blocked or silenced Clever-1, activating antigen presentation and promoting interferon gamma secretion by leukocytes. Additional pre-clinical studies have proven that Clever-1, encoded by the Stabilin-1 or STAB-1 gene, is a major source of T cell exhaustion and involved in cancer growth and spread. Observations from clinical studies to date indicate that Clever-1 has the capacity to control T cell activation directly, suggesting that the inactivation of Clever-1 as an immune suppressive molecule could be more broadly applicable and more important than previously thought. As an immuno-oncology therapy, bexmarilimab has potential as a single-agent therapy or in combination with other standard treatments including immune checkpoint molecules. Beyond immuno-oncology, it offers potential in infectious diseases, vaccine development and more.

Orna Therapeutics Announces First-in-Class, Breakthrough Data Demonstrating Potential of Circular RNA Platform at ASGCT 2022

On May 16, 2022 Orna Therapeutics, a biotechnology company pioneering a new class of fully engineered circular RNA (oRNA) therapies, reported that data from its lead isCAR program that validates the potential of the company’s novel oRNA technology and LNP delivery platform (Press release, Orna Therapeutics, MAY 16, 2022, View Source [SID1234614686]). Based on advances in the expression of oRNA as well as its delivery to immune cells, Orna has demonstrated tumor suppression and eradication in an animal model pointing to the possibility that oRNA-LNP based cancer therapies could eventually overtake cell therapies .

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Additional data to be presented demonstrate the utility of Orna’s proprietary FoRCE screening platform which has enabled the company to discover and characterize a major new resource for protein expression based on internal ribosome entry sites (IRESs). Orna will also present data showing the development of novel immunotropic lipid nanoparticles (LNPs), and the potential of oRNA in genetic muscle disease and vaccines. View full presentations from ASGCT (Free ASGCT Whitepaper) on our website here.

"At Orna, we’ve created the world’s leading circular RNA company and are building a platform and pipeline with the potential to change the way we treat disease," said Tom Barnes, PhD, Orna’s Chief Executive Officer and oral presenter at ASGCT (Free ASGCT Whitepaper). "Presented for the first time, these data suggest our oRNA technology and LNP delivery favorably combine to maximize our reach into multiple therapeutic areas – validating our expanded pipeline beyond cancer to include muscle genetic diseases and vaccines."

isCAR: Revolutionizing CAR-T Cell Therapy with the Possibility of Eradicating Tumors
Our lead program is an in situ CAR therapy that combines oRNA and custom engineered LNPs to create modified immune cells within the patient. This easily redosable format would not require patient lymphodepletion and would allow for reliable dose control, overcoming barriers of ex vivo CAR-T therapies. Data being presented demonstrate that oRNA-LNP can eradicate cancer cells in an animal model. Additional iterative data in rodents and non-human primates gives Orna confidence that this may successfully translate into humans.

"We are excited about the preclinical results in our lead isCAR program as we clearly see the opportunity to overcome significant hurdles in current ex vivo approaches, suggesting that oRNA-LNP based cancer therapies may eventually overtake cell therapies," said Robert Mabry, PhD and Chief Scientific Officer at Orna. "We believe that data from iterative animal studies can support our plans to deliver in situ CAR-T therapies to the clinic."

FoRCE: Formulated oRNA Cell-based Evaluation Platform
Orna is also presenting new data from our proprietary FoRCE screening platform, which captures the entire oRNA production, formulation, and evaluation process in an arrayed and automated format. In a first application, Orna has screened and characterized thousands of IRES elements in multiple primary human cell types. IRES identification and development is critical for optimizing oRNA function via tunable protein expression. Orna has discovered many novel IRES elements that drive oRNA expression to levels well above those of standard IRES elements, including some that show differential activity across cell types. These results open a new technological toolkit for driving protein expression from circular RNA.

Breadth of Platform
Orna has extended its oRNA-LNP technology into several other indications including Duchenne Muscular Dystrophy and vaccines and believes there are many additional opportunities this technology can bring to existing therapeutics.

Duchenne Muscular Dystrophy (DMD):
Orna will present data highlighting the ease of working with very large oRNAs. Data demonstrate, for the first time, non-viral delivery of a large, full-length, dystrophin-encoding RNA in human cells, as well as in vivo delivery of smaller length versions in mouse models. These data are an encouraging first step on the path to delivering full-length gene therapy to patients with DMD.

Vaccines:
Orna is investigating the suitability of oRNA combined with intramuscularly administered immunotropic LNPs for vaccine applications, including for COVID-19. The oRNA half-life observed in muscle and immune cells, combined with the intramuscular administration of immunotropic lipids, suggests that oRNA-LNP technology may be beneficially applied to vaccine development.

Conference presentation details are shared below.

Oral Presentations:
In situ CAR Therapy Using oRNA Lipid Nanoparticles Regresses Tumors in Mice
Presenter: Tom Barnes, Ph.D., CEO
Date/Time/Location: Monday, May 16, 2022 from 9:10 – 9:45 a.m. ET in Room 207
Session: Scientific Symposium: Function and Therapeutics Applications of Circular RNAs (circRNAs)

Discovery of Translation Initiation Elements Enabled by a Parallel Arrayed Screen of Full-length Viral UTRs in Synthetic Circular RNA
Presenter: Alexander Wesselhoeft, Ph.D., Director, Molecular Biology
Date/Time/Location: Monday, May 16, 2022 from 11:30 – 11:45 a.m. ET in Salon H
Session: Oral Abstract Session: Oligonucleotide Therapeutics

Poster Presentations:
Improved Immune Cell Expression with Circular RNA (oRNA) in vivo
Presenter: Kevin Kauffman, Ph.D., Principal Scientist
Date/Time/Location: Monday, May 16, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics I

Systemic Delivery of Circular RNA Encoding Partial Dystrophins and Expression in Skeletal Muscle
Presenter: Tatiana Fontelonga, Ph.D., Scientist
Date/Time/Location: Tuesday, May 17, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics II