This week in pharma… 19th August

ramarketing news round-up 

Chargepoint appoints new leadership, Bluebird gene therapy approved by FDA, and more!

ChargePoint Tech appoints new leadership

ChargePoint Technology designated Jonathan Bardsley as Global Applications and Solutions Engineering Manager. Jonathan will lead the A&SE (Applications and Solutions Engineering) department, supporting the sales and commercial team in improving the customer purchasing experience.

Jonathan’s role includes cooperating with the sales, marketing and service teams to guarantee customer feedback is implemented to improve and enhance the company’s proposal. 

Jonathan brings over 25 years of experience to the role and comes to the business with a unique perspective having previously been a ChargePoint Technology customer. 

“For many years I have worked closely with ChargePoint Technology as a loyal customer and I was always impressed by the outstanding service provided,” Jonathan commented. “That customer experience allows me to identify opportunities from which ChargePoint can continue to build and improve.” 

Bluebird gene therapy approved by FDA for rare blood disease

Zynteglo is cleared for transfusion-dependent beta thalassemia, but will come at a cost of $2.8 Million per patient.

The Food and Drug Administration on Wednesday approved a powerful new treatment for people with an inherited blood condition called beta thalassemia, clearing a personalized gene therapy developed by the biotechnology company Bluebird bio.

The therapy, which showed dramatic benefit in clinical testing, will come at a cost of $2.8 Million per patient, making it one of the most expensive medicines sold.

Its approval is a milestone in a number of other respects, too. Beta thalassemia, a disease that in severe cases requires regular blood transfusions for life, has for years been marked as a target for gene therapy. Bluebird’s treatment, which will be sold as Zynteglo, is the first of several in development to reach market in the U.S., giving patients an option that could free them from those transfusions and their associated side effects.

Tonix looks to live virus vaccines to prevent the spread of monkeypox

Tonix Pharmaceuticals claims that using a smallpox vaccine model could stop the spread of monkeypox.

The technology is being applied to vaccines against COVID-19, smallpox, and monkeypox. According to Seth Lederman, CEO of Tonix, the firm has “recreated a vaccine that we think is similar to what Edward Jenner used in 1798”.

While Lederman told us the undisclosed cell line is “pretty standard” in its technique, TNX-801 is a live virus vaccine based on synthesized horsepox (sHPXV) that it claims may lower toxicity and provide various safety advantages over existing vaccinia-based vaccines.

“Molecular techniques synthesize a virus, which is close to what we think [Jenner] used [and] we are growing it for human studies in a bioreactor,” said Lederman. “We think that that is a 

vaccine that could be widely used and potentially control smallpox in Africa where it is endemic.”

A universal drug target for ovarian cancer

The discovery of a blood vessel-promoting protein interaction found in all ovarian tumors may lead to a new druggable target for ovarian cancers.

Ovarian cancers are genetically and morphologically diverse and can be difficult to treat. However, these diseases all have an underlying commonality: they express the transcription factor PAX8. Researchers at the University of Pennsylvania School of Medicine recently discovered a protein interaction with PAX8 that regulates blood vessel development in ovarian tumors, which may present a druggable target for halting tumor growth.

“I was excited about the paper. PAX8 is a really exciting molecule, particularly as a target for high grade serous ovarian cancer,” said Joanna Burdette, a pharmaceutical scientist at the University of Illinois Chicago who was not involved in the study. “We really need a strategy to take that from being just a protein of interest … to something more clinically available.”

While PAX8 is commonly expressed in ovarian cancer tissue, clinicians use it primarily as a biomarker to track ovarian cancer cells. Its function is not well characterized, which makes it challenging to target on its own.

UK approves Moderna’s two-pronged COVID-19 booster

Britain’s drug regulator is the first public health authority to clear a vaccine designed to fight omicron.

British drug regulators on Monday approved a two-pronged vaccine from Moderna that’s designed to fight both the original version of the coronavirus and the omicron variant.

The decision makes the U.K. the first country to clear a COVID-19 booster that’s been specifically tailored to a newer form of the virus. While the original shots, like Moderna’s Spikevax, remain strongly effective at preventing hospitalization and death, they’ve had a harder time warding off infections from omicron and its subvariants.

Recipharm feature in PMPS August 2022

The article covers the rise of connected drug delivery and the challenges to come. With the ever growing demand for connected devices, what are the obstacles that must be overcome? Gemma Wood at Recipharm explores more, exploring the demand for self-administration and challenges in achieving drug connectivity.