SAN DIEGO--(BUSINESS WIRE)--Rejuvenate Bio, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M grant to complete IND-enabling activities for the development of RJB-0402 for the treatment of desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM)..

圣地亚哥--（商业新闻短讯）--Rejuvenate Bio今天宣布，加利福尼亚再生医学研究所（CIRM）已向该公司授予400万美元赠款，以完成IND授权活动，用于开发用于治疗desmoplakin基因变异性心律失常性心肌病（DSP ACM）的RJB-0402。。

RJB-0402 is a novel systemically administered investigational AAV8 gene therapy that drives liver specific expression of FGF21 protein, targeting multiple key pathological drivers of DSP ACM. Since the DSP gene is too large for gene replacement therapy, novel strategies are needed to treat DSP ACM. FGF21 has known salutary effects on cardiac dysfunction as well as ventricular arrhythmias, adipogenesis, inflammation, and fibrosis, all of which are DSP ACM hallmarks.

RJB-0402是一种新型的全身给药的研究性AAV8基因疗法，可驱动FGF21蛋白的肝脏特异性表达，靶向DSP ACM的多个关键病理驱动因素。由于DSP基因对于基因替代疗法来说太大，因此需要新的策略来治疗DSP ACM。FGF21对心脏功能障碍以及室性心律失常，脂肪形成，炎症和纤维化具有已知的有益作用，所有这些都是DSP ACM的标志。

Furthermore, clinical efficacy of RJB-0402 in DSP ACM may form the basis for the treatment in other forms of ACM..

此外，RJB-0402在DSP ACM中的临床疗效可能为其他形式的ACM治疗奠定基础。。

“DSP ACM is a rare, severe, life-threatening, and debilitating disease that typically manifests in young adults as a high risk of life-threatening ventricular arrhythmias, sudden cardiac death, and progression to heart failure,” said Dan Oliver, CEO & Co-Founder, Rejuvenate Bio. “There is currently no disease modifying therapy for patients with this disease, and our gene therapy RJB-0402 addresses a significant unmet medical need.”.

Rejuvenate Bio首席执行官兼联合创始人丹·奥利弗（DanOliver）表示：“DSP ACM是一种罕见的，严重的，危及生命的，使人衰弱的疾病，通常在年轻人中表现为危及生命的室性心律失常，心源性猝死和进展为心力衰竭的高风险。目前，尚无针对这种疾病患者的疾病缓解疗法，我们的基因疗法RJB-0402解决了严重未满足的医疗需求。”。

“The funding and strategic support from CIRM will accelerate development of our gene therapy candidate RJB-0402 into clinical trials for DSP ACM patients in desperate need of new therapies,” said Noah Davidsohn, Ph.D., Chief Scientific Officer & Co-Founder, Rejuvenate Bio. “We are confident that RJB-0402, a one-time gene therapy, could be a transformative regenerative medicine and the first disease modifying therapy to address the unmet medical need of DSP ACM patients, and the recent funding from CIRM will enable us to initiate our first in human clinical trial.”.

“CIRM的资助和战略支持将加速我们的基因治疗候选物RJB-0402的发展，使其成为迫切需要新疗法的DSP ACM患者的临床试验，”Rejuvenate Bio首席科学官兼联合创始人诺亚·戴维森（Noah Davidsohn）博士说。“我们有信心，RJB-0402是一种一次性基因治疗，可能是一种变革性再生医学，也是解决DSP ACM患者未满足的医疗需求的第一种疾病缓解疗法，CIRM最近的资助将使我们能够启动我们的首次人体临床试验。”。

Nonclinical proof-of-concept studies in the ACM mouse model showed that RJB-0402 significantly improved cardiac structure, function, and markedly reduced the arrhythmia burden, normalizing premature ventricular contractions (PVCs) to wild type levels. The clinical relevance of these efficacy signals provides a scientific rationale for further development as a therapeutic with the potential to meaningfully impact patients’ survival, symptoms, and quality of life..

。这些疗效信号的临床相关性为进一步开发治疗药物提供了科学依据，有可能对患者的生存、症状和生活质量产生有意义的影响。。

“Our goal is to move the most promising research forward,” said Dr. Abla Creasey, Vice President of Therapeutics Development at the California Institute for Regenerative Medicine (CIRM). “A one-time gene therapy treatment for patients with this rare cardiac disease DSP ACM, would have significant impact for patients with this degenerative disorder.

“我们的目标是推动最有希望的研究向前发展，”加利福尼亚再生医学研究所（CIRM）治疗开发副总裁Abla Creasy博士说。“对这种罕见心脏病DSP ACM患者进行一次性基因治疗，将对这种退行性疾病患者产生重大影响。

We look forward to supporting Rejuvenate Bio in bringing this regenerative therapy to patients with this degenerative disease.”.

我们期待着支持Rejuvenate Bio将这种再生疗法带给患有这种退行性疾病的患者。”。

About Desmoplakin Arrhythmogenic Cardiomyopathy (DSP ACM)

关于桥粒铂致心律失常性心肌病（DSP ACM）

DSP ACM is a severe, life-threatening, and debilitating, rare autosomal recessive disease that results in ventricular arrhythmias that are often life threatening and progressive cardiac dysfunction caused by fibrofatty replacement of ventricular myocardium which can lead to heart failure for which no disease modifying therapies exist.

DSP ACM是一种严重，危及生命且使人衰弱的罕见常染色体隐性遗传疾病，可导致室性心律失常，这些心律失常通常是由心室肌纤维脂肪替代引起的危及生命和进行性心功能不全，可导致心力衰竭，而不存在任何疾病缓解疗法。

While more than 10 different genes have been implicated in this heterogeneous disease, the majority involve one or more variants in desmosomal genes which are key components of the cardiac intercalated disc that are essential for the electrical and mechanical coupling of cardiomyocytes. These multiprotein complexes work together to provide cardiac mechanical integrity and strength as well as electrical communication and synchronization.

尽管这种异质性疾病涉及10多种不同的基因，但大多数涉及桥粒基因的一种或多种变异，桥粒基因是心脏闰盘的关键组成部分，对心肌细胞的电和机械耦合至关重要。这些多蛋白复合物一起工作以提供心脏机械完整性和强度以及电通信和同步。

While the exact prevalence of DSP ACM is unknown, it is estimated to affect an estimated 30,000-50,000 people in the United States..

虽然尚不清楚DSP ACM的确切患病率，但据估计，它会影响美国约30000-50000人。。

About Rejuvenate Bio

关于复兴生物

Rejuvenate Bio is a biotechnology company dedicated to developing novel gene therapies for chronic age-related diseases. Rejuvenate Bio has built a gene therapy pipeline with huge potential in chronic disease by utilizing clinically validated gene targets and a delivery approach that ensures well tolerated, durable expression.

Rejuvenate Bio是一家生物技术公司，致力于开发针对慢性年龄相关疾病的新型基因疗法。通过利用临床验证的基因靶标和确保良好耐受性，持久表达的递送方法，Rejuvenate Bio已经建立了一个在慢性疾病中具有巨大潜力的基因治疗管道。

Founded on scientific research developed at the Wyss Institute at Harvard Medical School, Rejuvenate Bio has developed groundbreaking therapies to treat chronic age-related disease in both humans and animals. The company is headquartered in San Diego, CA. For more information, visit www.rejuvenatebio.com..

Rejuvenate Bio以哈佛医学院Wyss研究所开展的科学研究为基础，开发了开创性的疗法来治疗人类和动物的慢性年龄相关疾病。该公司总部位于加利福尼亚州圣地亚哥。有关更多信息，请访问www.rejuvenatebio.com。。

About California Institute for Regenerative Medicine (CIRM)

关于加利福尼亚再生医学研究所（CIRM）

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission.

。

To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.

为了应对这一挑战，我们的团队由训练有素且经验丰富的专业人员组成，在动手创业的环境中积极与学术界和工业界合作，以快速跟踪当今最有前途的干细胞技术的发展。

With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality.

CIRM拥有55亿美元的资金和150多个活跃的干细胞项目，是世界上最大的致力于帮助人们实现细胞医学未来的机构。

For more information go to www.cirm.ca.gov.

有关更多信息，请访问www.cirm.ca.gov。