BHI News Section

Are you ready to dive into the world of groundbreaking biotechnology and the fight against chronic diseases? In this episode of BioTalk with Rich Bendis, we bring you an enlightening conversation with Dr. Nehal Mehta, Founder and CEO of Mobius Scientific, Inc. Dr. Mehta is not only a distinguished Professor of Medicine but also a visionary entrepreneur on a mission to revolutionize healthcare.

Join us as we explore the journey of Mobius Scientific, an early-stage biotechnology company with a transformative approach. Mobius is developing cutting-edge biologic therapies targeting the root cause of chronic diseases, starting with lipid accumulation. This innovative platform has the potential to change the lives of millions suffering from debilitating conditions.

Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy. 

Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. It is most common in African Americans and, while less prevalent, also affects Hispanic Americans. The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues. This mutation causes red blood cells to develop a crescent or “sickle” shape. These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events or crises can lead to life-threatening disabilities and/or early death. 

BALTIMORE, Dec. 7, 2023 /PRNewswire/ — BioGenCell, a pioneer in personalized cell therapy solutions, is excited to announce the launch of its fifth Phase II clinical trial site in the United States at the University of Maryland. The trial is focused on significantly reducing the need for amputation in patients with Chronic Limb-Threatening Ischemia (CLTI). The University of Maryland has joined this ambitious, global, placebo-controlled trial designed to offer transformative results for patients.

The prognosis for CLI patients is currently disheartening: within a year of diagnosis, nearly 20% of patients succumb to the disease while 30% will undergo amputation. Tragically, nearly 70% of these amputees do not survive beyond five years post-amputation.

WASHINGTON, Dec. 7, 2023 /PRNewswire/ — Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that it has acquired U.S. and Canadian rights to PONVORY^® (ponesimod) from Actelion Pharmaceuticals Ltd. (Janssen), a Johnson & Johnson Company. PONVORY^® is approved by the U.S. Food and Drug Administration (FDA) and Health Canada to treat adults with relapsing forms of multiple sclerosis (RMS), to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. PONVORY^® has a proven safety profile with over 10 years of data.

“The acquisition of Ponvory is a significant milestone for Vanda, as it expands our commercial portfolio and gives us access to a versatile immune response modifier that can potentially have broad application in treating a number of autoimmune-based disorders,” said Mihael H. Polymeropoulos, M.D., Vanda’s President, CEO and Chairman of the Board.

ARPA-H made its final hub location decision, selecting the historic Bowen Building in downtown Washington, D.C. as the site for its Stakeholder & Operations hub.

Why is this important? The new space will allow federal staff and contractors to work in one location, enhancing staff’s ability to manage programs and coordinate with federal partners—such as Congress, Health and Human Services, Centers for Medicare and Medicaid Services, U.S. Food and Drug Administration, the National Institutes of Health, and the Administration. Since the agency was authorized in March 2022, much of ARPA-H’s D.C. staff has been working remotely or from temporary offices.

SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.–(BUSINESS WIRE)–United Therapeutics (Nasdaq: UTHR), a public benefit corporation, today announced that the company has been named on Newsweek’s list of America’s Most Responsible Companies 2024. This award is presented by Newsweek and Statista Inc., the world-leading statistics portal and industry ranking provider. The list of awards was announced on December 6, 2023, is available on Newsweek’s website, and recognizes the top 600 most responsible companies in the United States, spanning 14 industries.

ROCKVILLE, M.D. and SUZHOU, China, Dec. 6, 2023 /PRNewswire/ — Innovent Biologics, Inc. (“Innovent”) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, metabolic, autoimmune, ophthalmology and other major diseases, today announces the licensing deal expansion with Synaffix B.V., a Lonza company (SIX:LONN) focused on commercializing its clinical-stage platform technology for the development of antibody-drug conjugates (ADCs) with best-in-class therapeutic index.

The expanded deal builds on a previous agreement signed in June 2021, under which Innovent is granted to deploy Synaffix’s ADC technologies on a target-specific, non-exclusive basis, including GlycoConnect®, HydraSpace® and one of its toxSYN® linker-payloads, for a therapeutic molecule (Innovent R&D code: IBI343, a CLDN18.2 ADC) which has advanced to Phase 1 clinical development.

A powerful web app to help drug designers accurately predict the pKa value of titratable residues

December 5, 2023, Baltimore, MD – ComputChem, an Early Charm company that develops computational chemistry software for drug design, has just launched its first product, iTitrate.

iTitrate is a web app that accurately predicts the pKa values of titratable residues by simulating an acid-base titration experiment. It computationally allows a protein to adjust its protonation and conformational states to solution pH. This state-of-the-art approach has been rigorously validated through references available on the ComputChem website.