ProQR Appoints Peter A. Beal, PhD, as Chief ADAR Scientist
ProQR Appoints Peter A. Beal, PhD, as Chief ADAR Scientist
LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 10, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today announced the appointment of Dr. Peter Beal, a professor of Chemistry at the University of California, Davis, as Chief ADAR Scientist. Dr. Beal, one of the top experts in ADAR and RNA biology and chemistry, has been a long-term collaborator of ProQR and a valued member of ProQR's Scientific Advisory Board, where he has played an important role in shaping the Company's ADAR-based RNA editing platform.
荷蘭萊頓 & 美國劍橋,2024年12月10日(環球新聞) -- ProQR Therapeutics NV.(納斯達克股票代碼:PRQR)(ProQR)是一家致力於通過其專有的Axiomer RNA編輯技術平台改變人們生活的公司,今天宣佈任命加州大學戴維斯分校化學教授彼得·比爾博士爲首席ADAR科學家。比爾博士是ADAR及RNA生物學和化學領域的頂尖專家之一,一直是ProQR的長期合作伙伴,也是ProQR科學顧問委員會的寶貴成員,在塑造公司的ADAR基礎RNA編輯平台方面發揮了重要作用。
Dr. Beal's decades of research at UC Davis have advanced the scientific community's understanding of ADAR-mediated RNA-editing mechanisms and their therapeutic potential. As Chief ADAR Scientist, Dr. Beal will expand on his existing contributions to ProQR by driving the development and optimization of the Company's ADAR editing platform, which aims to deliver life-changing treatments for patients with high unmet needs.
比爾博士在加州大學戴維斯分校的數十年研究推動了科學界對ADAR介導的RNA編輯機制及其治療潛力的理解。作爲首席ADAR科學家,比爾博士將通過推動公司ADAR編輯平台的開發和優化,拓展他對ProQR的現有貢獻,旨在爲有高未滿足需求的患者提供改變生活的治療方案。
"Pete has made tremendous impact on ProQR through our long-standing scientific collaboration and his contributions as a member of our Scientific Advisory Board. We are thrilled to now have him join the team in an expanded capacity as Chief ADAR Scientist," said Gerard Platenburg, Chief Scientific Officer of ProQR. "His deep expertise in ADAR biology and RNA chemistry, combined with his strategic insight, will be invaluable as we advance our pipeline of RNA-editing therapies."
「彼得通過我們長期以來的科學合作以及作爲我們科學顧問委員會成員的貢獻,對ProQR產生了巨大的影響。我們非常高興他現在以擴展身份加入我們的團隊,擔任首席ADAR科學家,」 ProQR首席科學官傑拉德·普拉特堡表示。「他在ADAR生物學和RNA化學方面的深厚專業知識,加上他的戰略洞察力,將在我們推進RNA編輯療法管線的過程中變得無價。」
Dr. Beal expressed his enthusiasm for expanding his role at ProQR, stating, "I have been deeply impressed by the vision, commitment, and progress of the ProQR team in harnessing RNA editing to address some of the most challenging genetic diseases. Having served on the Scientific Advisory Board, I've seen firsthand the transformative potential of ProQR's Axiomer RNA editing platform. I am excited to join the company in this new capacity at this pivotal time and to contribute to advancing RNA-editing science toward meaningful therapies for patients."
比爾博士表達了他對在ProQR擴展角色的熱情,他表示:「我對ProQR團隊在利用RNA編輯解決一些最具挑戰性的遺傳疾病方面的願景、承諾和進展深感印象。作爲科學顧問委員會成員,我親眼見證了ProQR的Axiomer RNA編輯平台的變革潛力。我很高興在這個關鍵時刻以新身份加入公司,併爲推進RNA編輯科學朝向患者的有意義治療貢獻力量。」
In his new role, Dr. Beal will oversee the platform optimization and scientific developments on the design of editing oligonucleotides to further strengthen ProQR's position as a leader in ADAR mediated RNA-editing innovation, drive preclinical development efforts, and expand the Company's therapeutic pipeline.
在他的新職位上,Beal博士將監督編輯寡核苷酸設計的 платформ оптимизации 和科學發展,以進一步鞏固ProQR作爲ADAR介導的RNA編輯創新領導者的地位,推動臨床前開發工作,並擴展公司的治療管線。
Peter A. Beal, PhD, biography
Peter A. Beal,博士,簡介
Peter A. Beal is a Professor in the Department of Chemistry at the University of California at Davis and current Director of the NIH-funded UC Davis Chemical Biology Graduate Program. Research from the Beal laboratory has advanced understanding of the structures and mechanism of action for ADAR enzymes responsible for adenosine to inosine RNA editing in humans. Recently, this knowledge has been applied to the design of guiding oligonucleotides capable of directing ADARs to make edits that correct disease-causing mutations in the transcriptome. Targets include mutations in mRNAs associated with Rett syndrome, Alzheimer's disease and Parkinson's disease. Dr. Beal teaches organic chemistry at the undergraduate level and several classes in nucleic acids chemistry and chemical biology at the graduate level.
Peter A. Beal是加利福尼亞大學戴維斯分校化學系的教授,以及當前由美國國立衛生研究院資助的UC戴維斯化學生物學研究生項目的董事。Beal實驗室的研究推進了對負責在人類中進行腺苷到次腺苷RNA編輯的ADAR酶的結構和作用機制的理解。最近,這些知識被應用於指導寡核苷酸的設計,使其能夠引導ADAR進行編輯,從而糾正轉錄組中導致疾病的突變。目標包括與Rett綜合症、阿爾茨海默病和帕金森病相關的mRNA突變。Beal博士在本科階段教授有機化學,並在研究生階段教授幾門核酸化學和化學生物學課程。
About Axiomer
關於Axiomer
ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer "Editing Oligonucleotides", or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
ProQR正在開創一種下一代RNA基礎編輯技術,稱爲Axiomer,這可能會爲多種疾病提供一種新型藥物。Axiomer「編輯寡核苷酸」的EONs以高度特異和針對性的方式介導RNA中單核苷酸的變化,使用一種存在於人類細胞中的分子機械,稱爲ADAR(腺苷脫氨酶)。Axiomer EONs的設計目的是招募和引導內源性表達的ADAR,將RNA中的腺苷(A)轉變爲肌苷(I)——肌苷被轉譯爲鳥苷(G)——將具有致病突變的RNA修正爲正常(野生型)RNA,調節蛋白質表達,或者改變蛋白質使其具有一種新功能,有助於預防或治療疾病。
About ProQR
關於ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer, which uses a cell's own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
ProQR Therapeutics致力於通過創造變革性的RNA療法來改變人們的生活。ProQR正在開創一種名爲Axiomer的下一代RNA技術,該技術使用細胞自身的編輯機制ADAR對RNA進行特定單核苷酸編輯,以逆轉突變或調節蛋白質表達,並可能爲有未滿足需求的罕見疾病和流行疾病提供一類新藥。基於我們獨特的專有RNA修復平台技術,我們正在以患者和親人爲中心擴大我們的產品線。
Learn more about ProQR at .
了解更多關於proqr的信息,請訪問 。
Forward Looking Statements
前瞻性聲明
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "continue," "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, express and implied statements regarding Dr. Beal's expected responsibilities and potential contributions to our therapeutic pipeline, the continued development, optimization and advancement of our Axiomer platform, the therapeutic goal of our Axiomer RNA editing oligonucleotides and product candidates, and the potential of our technologies and product candidates. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Eli Lilly and the Company; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
本新聞稿包含前瞻性陳述。除歷史事實的陳述外,所有其他陳述均爲前瞻性陳述,通常用諸如「繼續」、「預期」、「相信」、「可能」、「估計」、「期待」、「目標」、「打算」、「期待」、「可能」、「計劃」、「潛在」、「預測」、「項目」、「應該」、「將會」、「會」等類似的表達來表示。此類前瞻性陳述包括但不限於對Beal博士預計責任和對我們治療管線潛在貢獻的明示和暗示的陳述,Axiomer平台的持續發展、優化和提升,我們Axiomer RNA編輯寡核苷酸和產品候選的治療目標,以及我們的技術和產品候選的潛力。前瞻性陳述基於管理層的信念和假設,以及管理層在本新聞稿發佈日期可獲得的信息。我們的實際結果可能會因多種原因與這些前瞻性陳述所表達或暗示的結果有重大差異,包括但不限於我們向證券交易委員會提交的文件中所列的風險、不確定性及其他因素,包括我們在20-F表格中提交的年度報告的某些部分。這些風險和不確定性包括但不限於,前臨床研究和臨床試驗及我們與合作伙伴的其他開發活動的成本、時間和結果,而我們的運營和活動可能由於全球市場的供應和物流壓力而減緩或中斷;我們的預臨床和臨床項目在給定的時間表上啓動和執行的可能性,及依賴我們的醫藥外包概念的可靠性,以及及時招募受試者和患者以推進我們的臨床試驗和維持自身運營的可預測性;我們對代工廠商提供研究和開發物資的依賴及代工廠商供應中斷的風險;未來數據可能會改變早期臨床試驗初步結果的潛力;對申請或批准的監管審查的持續時間和結果的不可預測性,這些都是啓動並繼續推進我們的臨床項目所必需的;獲得、維持和實現與合作伙伴的合作的預期利益的能力,包括與禮來公司的合作;可能有損害、獲取知識產權的能力,以及獲取知識產權的成本;可能在研究和開發中生成的新數據可能出現的安全性或有效性問題;一般業務、運營、財務和會計風險,以及與第三方訴訟和爭議相關的風險;以及與全球經濟發展、地緣政治不穩定和衝突導致的宏觀經濟條件和市場波動相關的風險。鑑於這些風險、不確定性及其他因素,您不應對這些前瞻性陳述過度依賴,我們不承擔更新這些前瞻性陳述的任何義務,即使未來有新信息可用,法律要求的情況除外。
ProQR Therapeutics N.V.
ProQR Therapeutics N.V.
Investor contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Peter Kelleher
LifeSci Advisors
T: +1 617 430 7579
pkelleher@lifesciadvisors.com
投資者聯繫:
莎拉·凱利
ProQR Therapeutics N.V.
電話:+1 617 599 6228
skiely@proqr.com
或
彼得·凱勒赫
LifeSci顧問
電話:+1 617 430 7579
pkelleher@lifesciadvisors.com
Media contact:
Robert Stanislaro
FTI Consulting
T: +1 212 850 5657
robert.stanislaro@fticonsulting.com
媒體聯繫人:
羅伯特·斯坦尼斯拉羅
FTI諮詢
電話: +1 212 850 5657
robert.stanislaro@fticonsulting.com
譯文內容由第三人軟體翻譯。