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From South Korea to NYC: How One Company is Establishing a Global Presence to Develop RNA-Based Therapeutics for Alzheimer’s Disease and Other Neurodegenerative Diseases


New York is the global business hub that can open numerous doors for BIORCHESTRA.

Biorchestra was recently named the Awardee in the NYC Innovation QuickFire Challenge on Neuroscience, receiving $25,000 in grant funding; one year of residency at JLABS @ NYC - with the use of a bench, workstation and access to the JLABS @ NYC community; and mentorship from experts across the Johnson & Johnson Family of Companies. We sat down with the team to explore their science, their plans for leveraging the award, and why it is important to them to establish a presence in New York City.


Can you tell us about the journey of developing Biorchestra, from ideation to implementation?

Alzheimer’s disease has been known to have a genetic component, and there has been a profound amount of literature reporting the association of microRNAs with the disease. However, no efforts have been made to translate these results from the bench into the clinic. Our researchers believe that targeting the alterations that occur at the epigenetic level will be the most efficient way to develop disease-modifying therapies that can slow or halt progression of neurodegenerative diseases. To put this into action, we first discovered a disease-associated microRNA that was significantly upregulated in Alzheimer’s disease patient samples. Next, we conducted gain-of-function studies and discovered that this overexpressed microRNA may be the underlying cause of Alzheimer’s disease and decided to develop an anti-miRNA (antisense oligonucleotide) with a proprietary drug delivery technology to allow high brain cell-selective delivery. We expect to file for an IND with the FDA in 2022 for our lead therapeutic candidate, BMD-001, and enter clinical trials within the next 1-2 years. 


How is Biorchestra aiming to address an unmet need?

BIORCHESTRA’s BMD-001 targets a microRNA thought to be the cause of Alzheimer’s disease by intravenously administering a microRNA inhibiting antisense oligonucleotide to individuals that display an overexpression microRNA profile and has been specifically developed to allow enhanced delivery into brain cells. Furthermore, it has been designed such that following IV administration of BMD-001, the quantification of plasma or saliva microRNA concentrations can be used to predict the therapeutic efficacy of the drug. Future clinical studies will be necessary to prove the relationship between clinical measures, such as cognitive scores and the microRNA biomarker. In other words, we aim to advance:

  1. Companion and early diagnosis through the utilization of biomarkers
  2. Disease-modifying therapy by targeting the leading cause of Alzheimer’s disease
  3. Enhanced safety and patient-friendliness by injection via the intravenous route
  4. Clinical trial strategy utilizing both clinical and biomarker measures

We expect that this fully integrated approach covering all phases of drug development could potentially mitigate many challenges associated with neurodegenerative diseases.


How did you hear about the NYC Innovation QuickFire Challenge on Neuroscience, and what inspired you to apply?

The QuickFire Challenge is a well-known program that many start-ups admire in South Korea, due in part to the Seoul Innovation QuickFire Challenge series. BIORCHESTRA is also a current residents at the JPOD @ Boston. We believe that our participation in the QuickFire Challenge and residency at JLABS @ NYC will allow us to receive concrete and practical assistance as we enter the next development phase.


What does it mean to you to be an awardee in this QuickFire Challenge on Neuroscience?

We believe we are the first group to develop anti-miRNA treatments for Alzheimer’s disease. Our lead therapeutic candidate, BMD-001, is an innovative therapeutic developed based on this potentially groundbreaking idea. While there has been a considerable amount of excitement, there have also been some concerns from experts. Being acknowledged by a well-known company with a global presence in neuroscience is especially encouraging for our team. We’re hopeful that this will also amplify the interest that BIORCHESTRA has received from numerous pharma and biotech companies.


What does this mean for the future of your potential solution? What’s next?

As a small South Korean biotech company progressing into clinical development, the opportunity to receive close mentorship from experts at the Johnson & Johnson Family of Companies is significant. We also hope that this opportunity will be one of the milestones to build a strong business relationship.


What are your plans for leveraging the award (funds, residency, mentorship)?

Given our plans to initiate clinical development in the US, global fundraising has always been an attractive option. Therefore, we believe that this is an excellent opportunity to reach out and connect with life science investors. In addition, by utilizing the immense resources provided by Johnson & Johnson Innovation - JLABS and other renowned research institutes worldwide, we hope to develop into a global start-up. We believe that our participation in this program will help us receive mentorship on various aspects of first-in-human clinical trials for Alzheimer’s disease and ALS, FDA regulatory affairs, and the tools needed to build a collaboration-friendly business model.


You are based in Korea and also have a presence at the JPOD @ Boston. Can you tell us a bit about why it was important for you to have a global presence, particularly with a new presence in NYC?

JLABS @ NYC is located at the center of a biotech-friendly ecosystem surrounded by renowned hospitals and research institutes. We believe a presence in NYC will help further our research on BMD-001 for clinical development, the discovery of novel targets leveraging our platform technology, and the development of subsequent clinical candidates. New York is the global business hub that will open numerous doors for BIORCHESTRA.


What are your thoughts on the NYC healthcare ecosystem when it comes to neuroscience innovation?

We hope to develop partnerships with companies that have an abundant amount of experience in neurodegenerative disease therapy and RNA therapy. Thanks to the NYC healthcare ecosystem, we can utilize the distribution network of well-established pharmaceutical companies and search for collaboration opportunities to out-license BMD-001 and Alzheimer’s disease diagnostics kit(miRStra®).

If you’d like to learn more about Biorchestra, please visit their website. If you’d like to learn more about Johnson & Johnson Innovation – JLABS, please visit our website.


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