Milan,   Italy, July 08   --   Genespire, in collaboration with researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), has today announced the publication of preclinical data supporting the potential of its liver-directed immune-shielded lentiviral gene therapy approach to treat methylmalonic acidemia (MMA), a severe inherited metabolic disorder. The findings, published in the Journal of Hepatology, show that a single systemic administration of a lentiviral vector encoding the MMUT gene led to sustained improvements in disease features in a validated mouse model of MMA, with effects lasting for the average lifespan of laboratory mice. Since the mice were treated when young, the study supports the durability of the gene therapy through postnatal growth and maturation of the liver. MMA is caused by a deficiency of methylmalonyl-CoA mutase, an enzyme that plays a critical role in the body’s metabolism of food. Its absence results in the accumulation of toxic metabolites that lead to recurrent metabolic crises, growth failure, neurological impairment, and multi-organ damage. In the study, researchers also treated mice with a dose containing an optimized MMUT transgene, thereby improving therapeutic efficacy. In the same mouse model of MMA, this version exhibited a dose-dependent improvement of metabolomic biomarkers, with gene transfer efficiency exceeding 80% of the liver. The study also highlights that genetically corrected cells in the liver may, over time, replace the diseased ones, suggesting that therapeutic efficacy may progressively improve even when starting at lower initial doses. “Together, these findings indicate that Genespire is on a clear path towards the long-term correction of metabolic diseases which impact the liver and other organs,” said Lucia Faccio, CEO of Genespire.  “We  believe our approach has the potential to translate into human health in the form of a single-administration treatment for patients with MMA, and are committed to continuing our efforts in bringing our lead asset for MMA, GENE202, to the clinic. We would like to extend our gratitude to everyone involved in the research project, especially those at the SR-TIGET.” “We are confident that this study, together with other previous studies from our group at SR-TIGET, provides a comprehensive pre-clinical data package enabling the initiation of clinical testing in pediatric patients affected by MMA” concludes Dr Alessio Cantore, group leader at SR-TIGET and Associate Professor at Vita-Salute San Raffaele University, who supervised and coordinated the study and signed it as senior author....

Mumbai , July 8: The wait for this season's ultimate  party  track is finally over. Tips Music has released its brand-new  Punjabi  single ' Naachi   Jaave ', featuring the dynamic duo of Paradox and Parmish Verma. Packed with infectious beats, a catchy hook, and electrifying  energy , the song has already become a hot favorite among music lovers. Whether...

New Delhi, July 8: The Poshan Tracker platform has achieved a major milestone by recording 8.93 crore beneficiaries across the country , strengthening the government’s efforts to improve nutrition monitoring and service delivery. The digital platform has become an important tool for tracking the progress of nutrition programmes and ensuring better coordination among frontline workers, beneficiaries, and implementing agencies. It...

Kolkata , July 8:  The upcoming edition of  TTF ,  India 's oldest and largest  travel  show network, will be held in  Kolkata  from  10–12 July  2026  at  Biswa Bangla Mela Prangan . Organised by  Fairfest Media , the event will bring together tourism boards,  travel  companies, hospitality brands, airlines, attractions, and  travel  technology providers from across  India  and overseas, offering...

Bhopal, July 8: The Madhya Pradesh Government is working to expand digital healthcare services to the remotest parts of the state under the Ayushman Bharat Digital Mission (ABDM) , with the aim of improving access to quality and affordable healthcare through technology. The initiative focuses on strengthening digital health infrastructure and connecting patients, healthcare institutions, and service providers through an...

Kendrapara, July 8 (UDN): In a unique blend of tradition and technology, the Indian Institute of Technology (IIT) Bhubaneswar is providing engineering support for the construction of the chariot for the upcoming Baladevjew Temple Rath Yatra at Ichapur in Odisha's Kendrapara district. Representational Image  The annual Rath Yatra, scheduled to commence on July 16 , will witness the procession of...

New Delhi, July 8: The Adani Energy project has marked a significant step forward in strengthening economic cooperation between Kenya and India, paving the way for a new phase of collaboration in the energy sector. The project is expected to enhance infrastructure development, support Kenya’s energy requirements, and deepen bilateral ties between the two countries. The initiative reflects the growing...

Bengaluru/ Manipal , July 8:   Manipal   Academy  of  Higher   Education , an Institution of Eminence Deemed to be University, and  Novo   Nordisk Global Business Services  have expanded their long-standing collaboration to strengthen India's capabilities in pharmacometrics, a specialised scientific discipline that applies data, modelling and simulation to support drug development and clinical decision-making. Under the collaboration, Novo...