Latest news with #MeiraGTx
Newsweek
16-05-2025
- Business
- Newsweek
The New Era of Life Sciences: The Future is Now
When we published our 2024 report, children suffering from Leber Congenital Amaurosis 4 (LCA4)—a severe inherited retinal dystrophy—had only hope that one day they might see the world. Today, thanks to an investigational gene therapy, we know that the 11 children who participated in the trials can, for the first time in their lives, discern a toy, recognize a human face and even spot a grain of sand. The biotech behind this achievement is MeiraGTx, which has now filed its therapy for approval under exceptional circumstances in the U.K., which would expedite the process. Stories like that of MeiraGTx illustrate the transformative power of gene therapies on patients. More broadly, they highlight the potential for rare diseases R&D to emerge as the principal driver of therapeutic innovation. "Rare diseases have become a critical area for exploring new treatment modalities because, in many cases, there is no existing therapy, or even a model. For patients who have no options, innovative approaches are important," shares Marc Dunoyer, the CEO of Alexion, the rare diseases subsidiary of AstraZeneca. Ironically, the very severity and uncommonness of these illnesses can also be a blessing, as it allows for bolder approaches by both researchers and regulators. Moreover, rare disease drugs have historically had higher approval success rates compared to other drugs. The Tufts Center for the Study of Drug Development found that orphan-designated drugs had a Phase-1-to-approval success rate of 17 percent, compared to 7.9 percent for non-orphan drugs. Benefiting from faster approval pathways and enhanced regulatory incentives, entrepreneurs in the field face better odds of innovating successfully. And when they do, the implications of their therapies go far beyond their original target. "Many of the advances seen today, such as gene editing and RNA-based therapies, originated in rare disease research before moving into larger indications," says Dunoyer. Arcturus Therapeutics, for instance, is addressing cystic fibrosis via a new delivery mechanism. "Our cystic fibrosis program is a prime example, where the ability to inhale mRNA safely could have a huge impact on the pharmaceutical industry. The rare disease field offers a shorter regulatory pathway to approval and a higher likelihood of success. This makes it an attractive space for evaluating next-generation technologies like mRNA therapeutics," shares Arcturus' CEO, Joseph Payne, as the company is expecting results from their Phase 2 trials in 2025. Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics. Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics. We can perform comprehensive genomic profiling to uncover the molecular basis of a tumor. This allows doctors to provide tailored treatments. Over time, this will enable us to move to a future where we understand the molecular drivers for cancer and can deliver truly personalized healthcare. Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina. Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina. Whether it is providing clarity for families dealing with rare genetic disorders or enabling rapid and precise cancer diagnoses, our goal is to ensure patients receive the answers they need when they need them. This report has been paid for by a third party. The views and opinions expressed are not those of Newsweek and are not an endorsement of the products, services or persons mentioned. Click here to download the full report
Yahoo
14-03-2025
- Business
- Yahoo
MeiraGTx and Hologen launch AI-backed gene therapy venture
MeiraGTx has struck a deal with Hologen Limited, an artificial intelligence (AI) company specialising in multimodal generative AI models, to advance its Parkinson's disease gene therapy programme. Hologen has committed up to $430m in funding to support the development and commercialisation of AAV-GAD, MeiraGTx's investigational gene therapy for Parkinson's disease. The AI company will provide $200m upfront and allocate up to $230m in additional capital to fully finance the programme. As part of the deal, the companies will set up a newly-formed joint company, Hologen Neuro AI Limited. Hologen Neuro AI will also oversee early-stage clinical programmes targeting the central nervous system, including an investigational gene therapy for genetic obesity dubbed AAV-BDNF. MeiraGTx will retain a 30% ownership stake in Hologen Neuro AI while leading all clinical development and manufacturing activities, as per the 13 March announcement. In addition, MeiraGTx and Hologen will enter into clinical and commercial manufacturing supply agreements, with Hologen also acquiring a minority stake in MeiraGTx's manufacturing subsidiary. MeiraGTx's AAV-GAD programme is currently preparing for a Phase III trial, which is expected to kick off in mid-2025. The gene therapy works by delivering a gene encoding the enzyme responsible for producing the neurotransmitter GABA, which plays a role in motor dysfunction associated with Parkinson's disease. In a Phase II trial (NCT05603312), AAV-GAD demonstrated statistically significant improvements in motor function and quality of life (QoL). The study showed an 18-point improvement on the Unified Parkinson's Disease Rating Scale (UPDRS) Part 3 for patients receiving the high-dose treatment at 26 weeks, along with positive results on the Parkinson's disease Questionnaire (PDQ-39), a quality-of-life assessment. According to MeiraGTx's CEO Alexandria Forbes, Hologen's AI models have already been used to analyse data from the Phase II trial, identifying disease-modifying changes in brain physiology. 'Our collaboration with Hologen has broad significance for MeiraGTx and for drug development for neurological disorders in general. Deploying Hologen's LLMs [large language models] on MeiraGTx's clinical data allows the characterisation of disease modification in the CNS with unprecedented fidelity,' Forbes added. Beyond its Parkinson's programme, MeiraGTx is advancing AAV-AIPL1, an investigational gene therapy for congenital blindness, toward potential regulatory approval in the UK. The New York-headquartered biotech hit the headlines in February 2025 after 11 children who were born blind from birth gained significant vision improvements following treatment with AAV-AIPL1. The company said it is also in talks with the US Food and Drug Administration (FDA) to explore accelerated approval pathways in the US. In October 2023, Sanofi invested $30m into MeiraGTx through a pricing offering of ordinary shares. MeiraGTx sold 4 million ordinary shares for $7.50 each, granting Sanofi access to data from some of its programmes, including in immunology and inflammation, central nervous disorders and glucagon-like peptide-1 (GLP-1) and other gut peptides for metabolic diseases. "MeiraGTx and Hologen launch AI-backed gene therapy venture" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
24-02-2025
- Health
- Yahoo
MeiraGTx seeks UK approval after gene therapy restores sight in children born blind
MeiraGTx is advancing its investigational gene therapy toward expedited approval in the UK after 11 children who were born blind from birth gained significant vision improvements following treatment. The AAV-AIPL1 therapy is designed to address severe sight impairment caused by mutations in the AIPL1 gene. This condition is a form of retinal dystrophy that leads to severe visual impairment from birth, with affected individuals typically only able to distinguish between light and dark. The gene therapy involves injecting functional copies of the AIPL1 gene into the retina using an adeno-associated viral (AAV) vector, aiming to restore retinal cell function and prevent degeneration. Results from four of the 11 children were published in The Lancet on 21 February. The first four children treated were aged one to three years with severe retinal dystrophy linked to mutations in the AIPL1 gene. Each child received the therapy in one eye through subretinal injection. Outcome measures included visual acuity assessments, functional vision evaluations, visual evoked potentials, and retinal structure imaging. Before treatment, the children's visual acuity was limited to light perception. At an average follow-up of three and a half years post-treatment, the treated eyes showed significant improvement, with visual acuity improving. In contrast, the untreated eyes' visual acuity deteriorated to unmeasurable levels. Additionally, objective tests confirmed enhanced visual function and electrophysiological assessments indicated increased visual cortex activity specific to the treated eyes. Imaging revealed better preservation of retinal structure in treated eyes compared to untreated ones. MeiraGTx's CEO Alexandria Forbes said: 'These improvements extended outside the meaningful effects on vision and result in life-changing benefits in all areas of development including communication, behaviour, schooling, mood, psychological benefits and social integration.' Following these positive outcomes, a second cohort of seven children received treatment in both eyes. All 11 children treated with AAV-AIPL1 have so far exhibited meaningful visual improvements. One child experienced cystoid macular oedema in the treated eye, which partially improved over time and did not hinder the overall visual gains. No other significant safety concerns have been reported. In light of these findings, MeiraGTx has engaged in discussions with the UK's Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit AAV-AIPL1 for expedited approval. The company is also in talks with the US Food and Drug Administration (FDA) to explore accelerated approval pathways in the US. If approved, AAV-AIPL1 would be eligible for a priority review voucher (PRV). While gene therapies hold promise for rare diseases, commercialisation remains a challenge. High development costs, complex manufacturing processes, and limited patient populations often deter investment. Earlier this month, Italian charity Telethon Foundation submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for a gene therapy targeting Wiskott-Aldrich syndrome after the withdrawal of its commercial partner. This marks the second time the foundation has stepped in to support an abandoned gene therapy, following its efforts with simoladagene autotemcel for ADA-SCID. The foundation claims to be the first charity worldwide to take responsibility for drug production and distribution, highlighting the financial and logistical hurdles in bringing gene therapies to market. The incidence of AIPL1-associated severe retinal dystrophy is estimated to be around one in every million live births. During a call with investors, Forbes stated that the company is exploring all options to ensure global access to AAV-AIPL1, noting 'strategic interest' in the therapy. "MeiraGTx seeks UK approval after gene therapy restores sight in children born blind" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
