DoH, M42 And PureHealth Collaborate With GEMMABio To Enhance Gene Therapies For Children With Spinal Muscular Atrophy Type 1

The Department of Health – Abu Dhabi (DoH) is collaborating with M42, PureHealth, and GEMMABio Therapeutics to enhance gene therapy access for rare diseases. This partnership was announced following a meeting at the BIO International Convention 2025 in Boston. The collaboration aims to establish research centres in Abu Dhabi, facilitating clinical trials and future gene therapy approvals for patients across the MENA region.

Dr. Mohamed Alameri from DoH highlighted the importance of this partnership in positioning Abu Dhabi as a leader in gene therapies for rare diseases. He stated, "Our partnership with GEMMABio marks a pivotal step in Abu Dhabi’s journey to become a global leader in developing gene therapies for rare diseases leveraging the Emirati Genome Programme." This initiative aligns with efforts to enhance innovation and personalised care.

Collaboration to Advance Gene Therapies for SMA1

The collaboration will focus on creating a robust gene therapy research ecosystem in Abu Dhabi. A joint venture between M42 and GEMMABio will establish a top-tier development and manufacturing organisation. This facility will support translational and clinical research activities, making Abu Dhabi a regional hub for gene therapy advancements.

Abu Dhabi's infrastructure supports this initiative through streamlined licensing, co-commercialisation processes, and tax incentives for biotech startups. These measures aim to position the Emirate as a global leader in life sciences innovation. The Health, Endurance, Longevity and Medicine (HELM) Life Science Cluster further enhances these efforts by providing necessary resources.

Dr. Jim Wilson of GEMMABio expressed optimism about the partnership's potential impact on global access to treatments for rare diseases. He remarked, "This is a transformational partnership—one that will play a pivotal role in unlocking global access to life-saving treatments for patients with rare diseases." The collaboration aims to align scientific innovation with ambitious goals to accelerate progress where needed most.

M42's CEO Dimitris Moulavasilis emphasised the role of AI and data-led ecosystems in advancing health solutions. He noted that this partnership extends the Emirati Genome Programme's impact into gene therapeutics targeting rare diseases. The focus remains on developing genomic insights to combat rare diseases effectively.

Expanding Genomic Capabilities

Abu Dhabi has made significant strides in genomics through the Emirati Genome Programme, sequencing over 800,000 genome samples. This achievement contributes to one of the world's most advanced national genomic databases. The Emirate continues pioneering efforts in administering cutting-edge gene therapies previously unavailable in the region.

Rashed Saif Al Qubaisi from PureHealth highlighted Abu Dhabi's growing influence as a health innovation centre. He stated that collaborating with GEMMABio reinforces their commitment to solving complex health challenges globally. PureHealth aims to contribute through integrated research capabilities aligned with DoH’s vision.

International Collaboration Efforts

A high-level delegation led by DoH visited the United States from June 15-21, 2025, focusing on knowledge exchange and investment opportunities. Key stakeholders included entities like Mubadala BIO, Masdar City, KEZAD, NYUAD, Khalifa University, MBZUAI, and startAD. This mission aimed at accelerating advanced health solution adoption through new agreements.

This strategic partnership underscores Abu Dhabi's commitment to advancing precision medicine and rare disease therapies. By investing in research talent and infrastructure, the Emirate strengthens its position as a regional leader in medical innovation.

With inputs from WAM

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