The Indo-US Organization for Rare Diseases (IndoUSrare) has announced the winners of Pitch4RARE, its virtual startup pitch competition held on December 9, recognizing innovative, patient-centered solutions aimed at advancing equitable care for people living with rare diseases.

In a statement, the organization said that following the evaluation of six shortlisted pitches by an eminent panel of judges, and based on alignment with IndoUSrare’s mission, GenoPhe Biotech Pvt Ltd and IUVA Labs were named winners of Pitch4RARE 2025. The organization cited their innovative approaches and strong potential to deliver meaningful, scalable impact for patients and families affected by rare diseases. Attox Research Laboratories received special recognition.

The competition brought together a diverse cohort of startups addressing critical gaps across the rare disease landscape. Other finalist companies included Mamidi Health, Multifly Lifestyle Private Limited, and EFA Sciences.

Pitch4RARE is a global innovation catalyst designed to identify, validate, and accelerate bold ideas, regardless of where they originate, that can meaningfully improve the lives of people living with rare diseases and move those ideas toward patients with urgency.

According to the statement, the initiative is grounded in the belief that transformative ideas in rare diseases are not confined to elite institutions, well-funded laboratories, or a single geography. Breakthrough insights, technologies, and business models can emerge from clinicians, scientists, caregivers, patients, students, and entrepreneurs anywhere in the world.

What is often lacking, the statement noted, is not ingenuity but “visibility, validation, mentorship, and a credible pathway” to scale. Pitch4RARE seeks to bridge that gap by identifying high-potential innovations early, providing expert feedback and strategic support, and connecting innovators with partners who can help translate ideas into real-world impact.

The contest draws inspiration from pioneers who challenged the status quo in rare disease drug development, including John F. Crowley, founder of Amicus Therapeutics, driven by a personal mission to cure Pompe disease, and Dr. Emil Kakkis, founder of Ultragenyx, who worked closely with affected families to address lysosomal storage disorders.

“Pitch4RARE aims to identify and empower the next generation of such leaders—individuals and teams with the courage, clarity, and commitment to turn urgency into action and innovation into hope for patients and families who cannot afford to wait,” the statement said.

Despite progress since the Orphan Drug Act of 1983, approximately 95 percent of rare diseases, now numbering more than 11,000, still lack FDA-approved treatment options, the statement added. Each year, Pitch4RARE convenes innovators, early-stage startups, industry experts, investors, and patient advocates from across geographies to evaluate breakthrough ideas in biotechnology, healthcare, and life sciences.

The competition is a core component of IndoUSrare’s broader mission to bridge hope across nations by accelerating innovation, fostering cross-border collaboration, and supporting patient-centered solutions that can be implemented across diverse healthcare settings, including in low- and middle-income countries.

“Pitch4RARE reflects the growing maturity of the global rare disease innovation ecosystem,” said Harsha Rajasimha, host of Pitch4RARE 2025 and Founder and Executive Chair of IndoUSrare. “The winning teams and finalists demonstrated a strong understanding of patient needs, feasibility, and scalability, factors that are essential for translating innovation into real-world impact.”

Aligned with IndoUSrare’s vision of building global pathways of collaboration to address unmet needs in rare diseases, Pitch4RARE was designed to go beyond a traditional pitch competition. Startups were assessed not only on scientific and technological novelty, but also on feasibility, scalability, and patient-centered design, reinforcing the organization’s commitment to ensuring that innovation leads to accessible and equitable outcomes worldwide.

Participating startups gained global visibility through an international virtual platform, expert evaluation by leaders across technology, healthcare, and life sciences, and networking opportunities with biopharma companies, academic institutions, and patient advocacy organizations.

“As part of IndoUSrare’s commitment to nurturing early-stage innovation, Pitch4RARE winning startups will receive a cash prize, mentorship from senior leaders in rare diseases, and in-kind support, including discounted services such as clinical trial-related software solutions and potential access to real estate or incubation space in Prince William County,” the statement said. “Winners will also receive visibility through press coverage and social media highlights from IndoUSrare. All finalists will receive free registration to the next Indo-US Bridging RARE Summit, enabling continued engagement with global experts, industry leaders, policymakers, and patient communities.”

“For rare disease ventures, progress depends as much on informed capital and partnerships as it does on science,” said Dr. T. Leela Srinath, Founder of GenoPhe Biotech Pvt Ltd. “Pitch4Rare created a meaningful space to connect with the right funding and collaborative ecosystem.” GenoPhe pitched its prescription nutraceutical, Chaklz, for drug-resistant epilepsy.

“Pitching our nutritional solution for phenylketonuria patients at the IndoUSrare Pitch4Rare event provided an invaluable platform to engage with industry experts,” said Vihang Ghalsasi, Founder of IUVA Labs. “Patients with rare diseases like PKU deserve accessible treatments and care, and initiatives like this directly address that critical unmet need.”

Pitch4RARE submissions were evaluated by a panel representing leadership across industry, technology, and healthcare, including Abhishek Mathur, PhD, MBA of Symphony Technology; Chidiebube C. of Emerging Technology Centers; Christiaan Engstrom of BLPN; Mahesh Narayanan, MS of MatchPlay; and Mandeep Kaur, MD, MS of Valion Consulting. IndoUSrare also acknowledged the guidance and support of Dr. Narendra Chirmule of Symphony Technology.

IndoUSrare expressed gratitude to Amgen for its support as the Champion Sponsor of Pitch4RARE 2025 and for its shared commitment to advancing innovation, collaboration, and equity in rare diseases.

Through initiatives such as Pitch4RARE and the Indo-US Bridging RARE Summit, IndoUSrare continues to build a globally connected, equity-driven ecosystem that brings together startups, industry leaders, policymakers, researchers, and patient communities to accelerate access to diagnosis, treatment, and long-term support for rare disease patients worldwide.

Looking ahead to 2026, IndoUSrare, together with the Global Organization for Rare Diseases, has outlined a Vision 2030 to ensure that rare disease research and orphan drug development efforts do not perpetuate inequities by excluding underrepresented populations in the United States, India, and globally.

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