▎WuXiEdited by Kant Content Team

Today, Be Biopharma, dedicated to the development of B-cell therapies, announced the achievement of multiple key milestones. The lead project BE-101 for the treatment of hemophilia B has entered the clinical development stage; meanwhile, the company announced the selection of a second drug candidate for the treatment of hypophosphatasia (HPP). Both projects are based on Be Biopharma's robust and efficient B-cell medicine (BCM) platform.This platform utilizes gene-editing technology to modify B cells, enabling them to continuously produce therapeutic proteins, ultimately developing drug candidates with durability, titratability, and repeatability without the need for preconditioning, holding the potential to become a “best-in-class” gene therapy.

The company's recent milestones include:

In May 2024, Be Biopharma announced that the IND application for its potential "first-in-class" B-cell therapy BE-101 had been approved by the FDA.BE-101 is designed to enable sustained production of steady levels of Factor IX by B cells without the need for preconditioning, offering durability, repeatability, and titratability.The first human clinical trial, BeCoMe-9, has begun recruiting patients. This is a multi-center, dose-escalation study aimed at evaluating the safety and preliminary efficacy of BE-101 in adult patients with moderate to severe hemophilia B. The FDA recently granted BE-101 Fast Track designation.

The company announced that BE-102 has been selected as a candidate drug for the treatment of HPP, a severe genetic disorder with extremely high unmet medical needs. It is characterized byALPLLoss-of-function mutations in genes lead to insufficient alkaline phosphatase (ALP) activity, causing underdevelopment and fragility in bones and teeth. Current approved therapies require multiple injections per week and are only limited to pediatric-onset forms. Previously presented data indicates,BE-102 has the potential to generate active ALP in vivo and continuously maintain ALP levels.

The company also announced that it has completed an $82 million financing round, supported by multiple venture capital firms and pharmaceutical companies, including ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol-Myers Squibb, and Takeda Ventures. The funds will be used to achieve clinical proof-of-concept for BE-101 and advance the development of BE-102.

Disclaimer: The content team of WuXi AppTec focuses on introducing the research progress in global biopharmaceuticals and health. This article is for information exchange only, and the views expressed in the article do not represent the position of WuXi AppTec, nor does it imply that WuXi AppTec supports or opposes these views. This article is not a recommendation for treatment plans. For guidance on treatment options, please visit a正规 hospital.

Copyright Statement: This article is from the content team of WuXi AppTec. Individuals are welcome to share it on their social media platforms, but unauthorized reproduction by media or institutions in any form to other platforms is prohibited. For authorization to reproduce, please reply with "reprint" on the WeChat Official Account of "WuXi AppTec" to obtain reprint guidelines.