News

 
Join us for an enlightening episode of BioTalk with Rich Bendis as we explore the pivotal role of patient registries in rare disease research and treatment. Our guest, Harsha Rajasimha, MS, Ph.D., CEO of Jeeva Informatics Solutions and Founder and Executive Chairman of IndoUSrare, shares his extensive experience in clinical genomics data science and precision medicine.

In this episode, Dr. Rajasimha introduces Jeeva Informatics Solutions and IndoUSrare, his non-profit organization. He discusses the importance of patient registries in accelerating rare disease research, the current obstacles in the field, and the potential solutions.

We discuss the role of governments in rare disease research and treatment and the potential for collaboration with non-profits in clinical trials. Finally, Dr. Rajasimha shares the goals of the upcoming Indo-US Rare Summit at the GMU Campus in Arlington, VA.

Tune in for valuable insights from a leader in rare disease research and patient advocacy.

Listen now via your favorite podcast platform

• Apple – https://apple.co/3Ibcjp9
  
• Google – https://bit.ly/3IbcyAz
• Spotify – https://spoti.fi/42z2SrG
• Amazon Music – https://amzn.to/3Ob9Sqw
• TuneIn – https://bit.ly/42TnDy7
• YouTube (Audio) – https://bit.ly/42A18OR

Scheduled for October 29-30, 2023, at the George Mason University Campus in Arlington, VA, the Indo US Bridging RARE Summit 2023 aims to unite the global rare disease community and raise awareness about these conditions. While rare diseases are increasingly understood in the United States and Europe, there remains a lack of awareness in other parts of the world, including India. By addressing this gap, the summit aims to make a positive impact on the lives of 1.5 billion people in India and the global population of 8 billion.

Bringing together leaders representing diverse stakeholders in rare diseases, the summit serves as a platform for knowledge-sharing, exchanging experiences, and fostering collaborations to address the challenges faced by rare disease patients and their families. The event covers crucial themes such as cross-border patient engagement, care pathways encompassing screening, diagnosis, and treatment options, digitization of rare diseases including registries and emerging markets, diversity, equity, and inclusion in the globalization of orphan drugs, orphan drug clinical trials, and regulatory pathways.

IndoUSrare, the organizing body behind the summit, is committed to connecting rare disease patients worldwide and prioritizing education, research, and engagement to enhance their quality of life.

For sponsorship opportunities and event information, interested individuals can contact summit@indousrare.or and visit https://www.indousrare.org/summit/. The Indo US Bridging RARE Summit 2023 is an important platform for collaboration and knowledge exchange to make a positive impact on the rare disease community worldwide.

The summit planning committee includes prominent individuals representing various organizations and institutions involved in rare disease research and advocacy. Some of the committee

ROCKVILLE, Md. , May 9, 2023 /PRNewswire/ — MaximBio® is proud to announce it has signed a new agreement with the National Institutes of Health (NIH) through the Rapid Acceleration of Diagnostics (RADx® ) initiative, continuing a relationship fostered during the height of the COVID-19 pandemic. Building on their previous collaboration which resulted in the successful launch of the MaximBio ClearDetect® COVID-19 Antigen Home Test, the new partnership is centered around development of a COVID-19 and Influenza A/B combo test employing novel Lateral-Flow Technology. This technology, paired with the innovative FiarFly™ analyzer platform, and MaximBio’s proprietary manufacturing techniques, results in class-leading assay performance.

Walking Fish to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform to engineer novel B-cell based medicines for the treatment of serious diseases

ROCKVILLE, Md., May 04, 2023 (GLOBE NEWSWIRE) — MaxCyte, Inc., (Nasdaq: MXCT; LSE: MXCT), a leading, cell-engineering focused company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell-based therapeutics and to support innovative, cell-based research, today announced the signing of a strategic platform license (SPL) with Walking Fish Therapeutics, Inc. a biotechnology company that is rapidly advancing B cell-based therapeutics.

Under the terms of the agreement, Walking Fish obtains non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform. In return, MaxCyte is entitled to receive platform licensing fees, clinical milestone payments and sales-based payments.

• Ground-breaking approval enables adults aged 60 years and older to be protected from RSV disease for the first time
• The approval is based on data from the positive pivotal AReSVi-006 phase III trial that showed exceptional efficacy in older adults, including those with underlying medical conditions, and in those with severe RSV disease
• US launch is planned before the 2023/24 RSV season

GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Arexvy (respiratory syncytial virus vaccine, adjuvanted) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. This is the first RSV vaccine for older adults to be approved anywhere in the world.

Tony Wood, Chief Scientific Officer, GSK, said: “Today marks a turning point in our effort to reduce the significant burden of RSV. Arexvy is the first approved RSV vaccine for older adults, expanding GSK’s industry-leading vaccine portfolio, which protects millions of people from infectious diseases each year. Our focus now is to ensure eligible older adults in the US can access the vaccine as quickly as possible and to progress regulatory review in other countries.”

BETHESDA, Md., May 03, 2023 (GLOBE NEWSWIRE) — Gain Therapeutics, Inc. (Nasdaq: GANX), a biotechnology company leading the discovery and development of allosteric small molecule therapies, today announced that Innosuisse, the Swiss Innovation Agency, supports GT Gain Therapeutics SA with the amount of CHF 2.5 million (~$2.8 million) to develop GT-02287, Gain’s lead program for GBA1 Parkinson’s disease.

The Company’s Swiss Accelerator innovation project supported by Innosuisse entitled “Harnessing Structurally Targeted Allosteric Regulators to treat Parkinson’s Disease” includes clinical pharmacology and preclinical studies with the Company’s drug candidate GT-02287, which will be conducted as the lead program progresses through Phase 1 and Phase 2 clinical studies in GBA1 Parkinson’s disease.

GBA1 mutations are the major genetic risk factor for Parkinson’s disease and are associated with earlier onset of the disease, faster disease progression and increased rates of cognitive decline. Mutations of the GBA1 gene cause misfolding and dysfunction of the enzyme beta-glucocerebrosidase (GCase). Preclinical models of GT-02287 have shown the ability of this orally bioavailable and brain-penetrant molecule to target and bind to GCase, prevent its degradation and allow its transport to the lysosomes where the enzyme can carry out its biological function. Furthermore, the data from preclinical models demonstrate that enhancement of lysosomal GCase activity by GT-02287 protects against key pathological features of Parkinson’s disease, including alpha-synuclein related pathology and an increase in the survival of dopaminergic neurons. These findings support the potential of GT-02287 to slow or halt disease progression in GBA1 Parkinson’s disease.

Registration is Open for the 2023 Children’s National Innovation Day & Showcase

Date: May 31, 2023 Time: 10:00 am – 7:00 pm Location: Children’s National Research and Innovation Campus (CNRIC), Washington, DC Registration Link: Click here to register

The organizers of the 2023 Children’s National Innovation Day & Showcase are thrilled to announce that registration is now open for this premier event highlighting innovation in pediatric healthcare. The event will take place on May 31, 2023, at the Children’s National Research and Innovation Campus (CNRIC) in Washington, DC.

Virginians have often sought experimental medical treatments outside the state — traveling to North Carolina, Boston or one of the Mayo Clinic’s locations. However, the Paul and Diane Manning Institute of Biotechnology at the University of Virginia aims to change that.

“We intend to tackle the biggest challenges in health care and to empower our researchers to make the life-changing breakthroughs that will transform care for people across Virginia and beyond,” says Dr. K. Craig Kent, CEO of UVA Health and executive vice president for health affairs at U.Va. “We’re on a cusp of a revolution in health care.”

Construction of the institute, which will be built in the 54-acre Fontaine Research Park adjacent to U.Va.’s Grounds, is set to be complete in 2026 or 2027. However, the university is forging ahead with hires, including about 100 scientists who will research potential treatments for diseases like diabetes and Alzheimer’s. Kent anticipates employing 1,000 to 1,400 people at the institute, which will include 30,000 to 40,000 square feet of lab space and a biomanufacturing facility.