BioHealth Innovation Weekly for November 07, 2023

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November 7, 2023

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Unlocking the Future of Stem Cell Research with MSCRF: A Conversation with Ruchika Nijhara, PhD, Executive Director of Maryland Stem Cell Research Fund (MSCRF)

Are you curious about the world of transformative regenerative medicine and stem cell research? Are you eager to understand how Maryland champions researchers and companies in the field of stem cell and regenerative medicine? Do you want to learn more about the Maryland Stem Cell Research Fund (MSCRF)? Listen to the latest episode of BioTalk with Rich Bendis as he welcomes MSCRF Executive Director, Ruchika Nijhara, PhD.

In this enlightening episode, Dr. Nijhara, a forward-thinking leader driven by her passion for advancing scientific innovation, delves into the funding mechanisms that Maryland employs to support research and development at all stages and fosters collaboration and innovation.

QIAGEN and Element Biosciences partner to offer complete next-generation sequencing workflows for the AVITI System

Partnership combines QIAGEN’s QIAseq panels, CLC LightSpeed, and QCI Interpret software to support Element’s AVITI System users in genomic analysis
Collaboration to accelerate discovery, enhance cost efficiencies, and improve turnaround times for genomic applications in the scientific community
QIAGEN to present performance data for universal QIAseq library preparation panels running on other sequencing platforms at ASHG Annual Meeting 2023 in Washington, DC

Venlo, the Netherlands, and San Diego, California, Nov. 02, 2023 (GLOBE NEWSWIRE) -- QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) and Element Biosciences, Inc. today announced a strategic partnership to offer comprehensive next-generation sequencing (NGS) workflows for the Element AVITI™ System, an innovative sequencing platform.

Element’s AVITI System is a versatile benchtop sequencer offering a combination of performance, cost, and flexibility for a wide range of NGS applications. For customers using the AVITI System, QIAGEN provides Sample to Insight NGS workflows with validated QIAseq panels and integrated bioinformatic solutions, including CLC LightSpeed and QCI Interpret software.

“The collaboration between QIAGEN and Element Biosciences delivers unprecedented insights across various genomic applications to our customers worldwide. Researchers are increasingly searching for complete solutions and workflows from sample to insight, and we are committed to advancing the field together by providing innovative technology and applications that help researchers accelerate discovery,” said Nitin Sood, Senior Vice President, Head of Life Sciences Business Area at QIAGEN.

Indo US Bridging Rare Summit Heralds a New Era of Cooperation for Rare Diseases Orphan Drugs Development

The FDA’s Commissioner, Dr. Robert M. Califf, recently visited India and shared his opinion on India’s Unique Opportunity and Important Responsibility as the Pharmacy to the World. The Inaugural 2-day Indo US Bridging RARE Summit was held on Oct 29 and 30, 2023 at George Mason University in Arlington, VA. The organizing committee led by Dr. Harsha Karur Rajasimha at the Indo US Organization for Rare Diseases (IndoUSrare), a US-based nonprofit committed to combating rare diseases, are aiming to catalyze this potential cooperation and harmonization between the FDA and Indian regulators to create a pathway for biopharmaceutical sponsors to start commercially launching orphan therapies in India. The effect can be far reaching, saving or impacting millions of lives. Dr. Peter Marks, Director of Center for Biologics Evaluation Research (CBER), FDA, keynote speaker at the Summit, announced the Creation of Operation Warp Speed for Rare Diseases, a pilot program called Support for Clinical Trials Advancing Rare Disease Therapeutics (START).

(HERNDON, Va.) October 24, 2023: The launch of Operation Warp Speed in 2020 to develop COVID-19 vaccines was a resounding success, developing and making available the vaccines to the general public in record time and saving an estimated 3.2 million lives and $1.15 trillion in the U.S. alone.1 As a result of that achievement, the U.S. Food and Drug Administration (FDA) is launching a pilot program — Support for Clinical Trials Advancing Rare disease Therapeutics (START) — this year to accelerate the pace of development of therapeutics for very small populations with very high medical need. 1 Globally, rare diseases are defined by The World Health Organization (WHO) as a disorder which affects less than 6.5 to 10 people out of 10,000.2 According to a report by RARE-X, there are over 10,897 known rare diseases affecting over 400 million people worldwide. Since the Orphan Drug Act of 1983, the FDA has approved about 1100 orphan drugs to treat about 5-7% of rare diseases. Hence, a majority of rare diseases remain without treatment options. At the recently concluded Indo US Bridging RARE Summit 2023, Dr. Peter Marks, director at the FDA’s Center for Biologics Evaluation and Research (CBER), spoke about the START program, the India opportunity, and stated, “We have fifteen FDA approved gene therapies till date. We are very committed to working with the rare disease communities for gene therapies and with our colleagues globally. India is an incredible opportunity in terms of capacity and capability. By leveraging them and working together, we can achieve some wonderful things for people with rare diseases.”

United Therapeutics to Acquire Miromatrix Medical

October 30, 2023 06:00 AM Eastern Daylight Time - SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C. & EDEN PRAIRIE, Minn.--(BUSINESS WIRE)--United Therapeutics Corporation (Nasdaq: UTHR) and Miromatrix Medical Inc. (Nasdaq: MIRO) announced today a definitive agreement for United Therapeutics to acquire Miromatrix.

Miromatrix is a life sciences company focused on the development of bioengineered organs composed of human cells. United Therapeutics is a biotechnology company with six FDA-approved therapies to address rare, life-threatening conditions, and a pipeline that includes four ongoing registration-phase studies. The acquisition of Miromatrix will expand United Therapeutics’ existing complementary platform of organ manufacturing programs, which include ex-vivo lung perfusion, xenotransplantation, 3-D bioprinting, and regenerative medicine approaches with the objective of creating an unlimited supply of tolerable, transplantable organs.

ATCC and USP Launch First Set of Products to Advance Quality and Reduce Risk in the Development of Biological Therapeutics and Vaccines

Six new products that can be used to detect residual host cell genomic DNA (gDNA)

MANASSAS, Va. and ROCKVILLE, Md., Oct. 30, 2023 /PRNewswire/ -- Today, ATCC and U.S. Pharmacopeia (USP), two premier materials management and standards organizations, announced the launch of their first set of joint products to advance the quality control and reduce the risk in the manufacturing of biological therapies and vaccines. This initial set of six products consists of highly characterized and pure genomic DNA (gDNA) from cell lines used in bioproduction that can be used to measure residual host cell DNA in various biotherapeutic products, as required by regulatory authorities. The presence of gDNA is a predominant concern in the development of biological therapies as it can pose a safety risk if it is not removed from the product.

"Developing cutting-edge medicines in the biologics space requires trusted reference materials and standards to ensure quality, consistency and safety," said Amanda Cowley, General Counsel and SVP Legal, Strategy and People of USP. "With increased demands around the globe for new biological medicines on accelerated timelines, it's essential that there are products and solutions that can be used to address and advance quality assessment and bring confidence to the development process. By focusing on the detection of gDNA, this first set of ATCC and USP products is significant for the future of biological development as it addresses a common concern when it comes to quality and mitigating risk."

Vaccitech Renames as Barinthus Biotherapeutics to Highlight Strategic Evolution into a T Cell Immunotherapy Company Targeting Chronic Infectious Diseases, Autoimmunity, and Cancer

OXFORD, United Kingdom, Nov. 06, 2023 (GLOBE NEWSWIRE) -- Barinthus Biotherapeutics plc (NASDAQ: BRNS), formerly Vaccitech plc (NASDAQ: VACC), a clinical-stage biopharmaceutical company developing novel T cell immunotherapeutic candidates designed to guide the immune system to overcome chronic infectious diseases, autoimmunity and cancer will present data from its Phase 2 Hepatitis B trials of VTP-300 at The American Association for the Study of Liver Diseases (AASLD) – The Liver Meeting® 2023, November 10-14 in Boston, MA.

The company also announced today that it has changed its name to Barinthus Biotherapeutics plc to represent the evolution and expansion of its focus beyond vaccines. The company expects to begin trading under its new name and ticker (Nasdaq: BRNS) effective as of November 7, 2023. As part of the rebranding, the company has also updated its website -- learn more at www.barinthusbio.com.

Johnson & Johnson Innovation Names Awardees in the Advancing Oncology InnoVAtion QuickFire Challenge

October 25th, 2023 – Johnson & Johnson Innovation, in collaboration with Carilion Clinic Innovation, Verge Alliance (with support from City of Roanoke), and Virginia Tech Corporate Research Center (VTCRC), announced today at the Harnessing the Potential of Antibody Drug Conjugates (ADC) in Oncology event at JLABS @ Washington D.C. that Bacchus Therapeutics, Luminary Therapeutics, and QurCan Therapeutics Inc., have been selected as the awardees in the Advancing Oncology InnoVAtion QuickFire Challenge.

The challenge invited innovators from across the globe with an active interest in the Virginia innovation ecosystem to submit potential solutions aiming to transform patient outcomes in oncology in adult populations with potential applications in pediatric oncology. The awardees hope to nurture Virginia’s outstanding innovation ecosystem and spearhead innovative solutions to enhance the quality of life for cancer patients across the globe.

BioBuzz: Addimmune Prepares to go Public, Initiate Phase Ib HIV Trial

By Alex Keown | October 31, 2023 -

Addimmune, currently American Gene Technologies HIV-focused research, is expected to become a publicly traded company in early 2024 following a merger with 10X Capital Venture Acquisition Corp. III, a special purpose acquisition company (SPAC).

Following the merger with the SPAC company, Addimmune will use the raised funds to support its planned Phase Ib clinical trial assessing the company’s Gag-specific AGT103-T, a genetically modified cell product, CEO Jeff Galvin told BioBuzz. The merger announcement comes months after plans to separate the HIV business were shared by AGT.

Addimmune launched with the goal of building on the success of a Phase I study assessing AGT103-T as a potential therapeutic for HIV that could offer a functional cure of the disease. Trial data revealed that 100% of participants showed active immune responses to HIV after participants ceased taking their antiretroviral therapy. The data showed that several of the seven trial participants achieved significant viral suppression, which signals the possibility that AGT103-T has the potential to deliver a functional cure to HIV patients. The Phase I data was published in November 2022 in Frontiers in Medicine, a peer-reviewed scientific journal focused on medical advancement.