With its proposal for a potential treatment for rare diseases like gene therapy, sickle cell, initial investors are losing in high-ambitioned areas such as obesity and cancer, as sales for some new remedies decrease.
In the previous year, some drug manufacturers have withdrawn from the region, including the fugger, which has recently stopped selling their gene therapy for Hemophilia, priced at $ 3.5 million per patient. Bluered bio, a gene therapy pioneer was sold at about $ 10 billion once, private equity firms in $ 30 million last month.
In 2024, the developers of gene therapy and gene-editing products picked up less than $ 1.4 billion in the 39 vertebra round, according to a data analysis Delforma For Roots,
In 2023, he raised $ 3.5 billion in 60 deals, which was 57% below the $ 8.2 billion summit of the sector in 122 deals in 2021.
To return to investor interest in gene therapy, “There are better ways to make some of these complex products, cheap ways,” said Sabin Bral, Ernst and Young Global Life Sciences Deal Leader.
Companies like Novartis say they are continuing gene therapy research. More than 95% of infants born in the US with a rare neuromuscular disorder called spinal muscular exploitation are now treated with its zolgensma gene therapy and is being developed for older children.
Chief Operating Officer Stuart Arbakal said that vertex pharmaceuticals continue to see patients with sickle cell diseases starting the process to get their causing therapy.
He said that treatment, which was only $ 10 million sales of 2024, was followed as the company believed that gene editing introduced the best solution for people living with weak disease.
Gene therapy-a one-time process of inserting a modified gene to compensate for a defective genes or changes that the patient’s cells produce protein-a complex process in which patients have often to be hospitalized, not always clear with insurance coverage.
The US Food and Drug Administration has supported success treatments, which have multimilian-dollar prices.
Peter Marx, director of the FDA’s Center for Biological Evolution and Research, said that Reuters believe that scientific challenges, manufacturing difficulties and genuine the therapy applications will be worked on “speed bumps” including the real world application of gene therapy.
Marx said earlier this month, “We need to combine a streamlined regulatory process (…) that there is a package of things to reduce costs with manufacturing reforms so that they are no longer the cost prohibitory.” “I hope we have a group that is going to come here through the next years.”
Safety issues are also a matter of concern even after approval. Sarpta Theraputics said that a 16 -year -old boy died this week after a rare muscle dystrophy of the company died of acute liver failure after receiving the gene therapy.
Meanwhile, weight -loss drugs are expected to look at an annual sales of $ 150 billion in the coming years, as new, highly effective drugs have increased external demand. Obesity medical science attracted $ 1.75 billion in enterprise capital last year, about $ 630 million in Triple 2023.
Overall, the global bioforma venture funding increased from $ 23.2 billion in 2023 to $ 23.2 billion in 2023. According to cancer, the top area was $ 10.3 billion. Deelforma.
