The U.K. health technology assessment body has given its approval to the use of nine digital health products for treating mental disorders, enabling them to get conditional reimbursement in the National Health Service (NHS), while real world evidence of their cost effectiveness is collected.
Sania Therapeutics Inc. is setting out its stall at the American Society of Gene & Cell Therapy (ASGCT) conference in Los Angeles this week, after generating proof of concept for its chemogenetics approach to treating motor disorders. The company has engineered adeno-associated viral vectors that can be targeted to specific cell types. It will use these to deliver well-characterized ion channels to dysfunctional motor neurons. The ion channels will then be selectively controlled by an activating drug, which is taken orally.
Regulatory T-cell specialist Dualyx NV has closed a €40 million (US$43.5 million) series A to progress the lead autoimmune disease program to the clinic and to take forward two other Treg-based therapies. The company brings together expertise in antibody design with understanding of the role Tregs play in supressing the immune response to maintain homeostasis and self-tolerance, preventing autoimmunity.
A new mouse model of an inherited form of dystonia has shown the spinal cord is the driver of the condition, overturning previous understanding that the movement disorder is caused by disruption of neural circuits in the brain. The connection was demonstrated by selectively deleting torsin family 1 member A (TOR1A), the gene that causes dystonia, in the neurons of the spinal cord only.
The U.S. Inflation Reduction Act (IRA) may look like domestic affair, but the drug price controls it is bringing in are set to impact the biopharma sector across the globe.
The U.S. Inflation Reduction Act (IRA) may look like domestic affair, but the drug price controls it is bringing in are set to impact the biopharma sector across the globe.
The industry is not over the post-COVID-19 funding crash, and as the dust settles there are mixed signs for future prospects, with some metrics in decline, others more or less back to pre-pandemic levels, and some showing signs of improvement. But on the key productivity metric, there is a downward trend, with fewer new molecular entities (NMEs) approved by both the U.S. FDA and EMA over the last year and a half.
The IPO market in Europe is firmly shut and not a single company went public in the first quarter of 2023. The impact of this is trickling down to limit access to venture capital for biotechs.
A base-by-base comparison of the genome sequences of 240 species of mammals has pinpointed sites in the human genome where mutations are likely to cause disease. The sites are all perfectly conserved across the mammalian family tree over 100 million years of evolution, indicating they underlie fundamental biological processes that do not tolerate diversity or change very well.