After years of lab work, patients with Alzheimer’s disease are set to receive a vaccine that stimulates the immune system in the hope it will slow or even prevent the disease.

The first-in-human trial run by the Brigham and Women’s Hospital (BWH) in Boston, Massachusetts, will involve an intranasal vaccine using the immune modulator Protollin, an investigational intranasal agent that stimulates the immune system.

The phase 1 trial will determine the safety and tolerability of the nasal vaccine as well measure the effect on participants’ immune response, including its effects on white blood cells by examining cell surface markers, gene profiles and functional assays, says BHW.

“For 20 years, there has been growing evidence that the immune system plays a key role in eliminating beta amyloid,” said Professor Tanuja Chitnis from BWH and principal investigator of the trial.

Composed of proteins from bacteria, Protollin is designed to activate the white blood cells in the lymph nodes on the sides and back of the neck, which researchers hope will then migrate to the brain and trigger the clearance of beta amyloid plaque, one of the hallmarks of Alzheimer’s disease. Protollin has been used safely in humans as an adjuvant for other vaccines.

The US Food and Drug Administration (FDA) has finally accepted bluebird bio’s gene therapy beti-cel (betibeglogene autotemcel) for priority review after years of setbacks.

The one-off gene therapy is for adult, adolescent and paediatric patients with beta-thalassemia across all genotypes who require regular red blood cell (RBC) transfusions.

If approved, the therapy – known as Zynteglo in Europe – will be the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia.

Beta-thalassemia is a severe genetic disease where mutations cause significantly reduced haemoglobin levels, which can result in severe anaemia and lifelong dependence on blood transfusions.

Transfusions do not address the underlying genetic cause of the condition and can have serious side effects.

By adding functional copies of a modified form of the mutant gene into a patient’s own haematopoietic stem cells, beti-cel (pronounced BEH-tee-cell) can correct the haemoglobin deficiency that causes the condition.

The FDA priority review ruling is based on data from two phase 3 studies of the therapy, Northstar-2 and Northstar-3, as well as a long-term follow-up study LTF-303.
  
Together the studies represent more than 220 patient-years of experience, following 63 paediatric, adolescent and adult patients for more than seven years.

The UK life sciences sector has received another boost with the official unveiling of AstraZeneca’s (AZ) new £1bn research and development facility in Cambridge.

The facility will house 2,200 research scientists in 19,000m2 of laboratory space featuring the most advanced robotics, high-throughput screening and AI-driven technology.

The Discovery Centre will support AstraZeneca’s focus on specialised and precision medicines and foster the development of next-generation therapeutics, including nucleotide-based, gene-editing and cell therapies, said the company.

It will also play an important role in AZ’s R&D network, which already includes strategic research centres in Sweden and the US and development facilities in China and Japan. Much of the $7bn that AZ invests annually on R&D will be spent in the UK.

The disc-like structure is also ‘a feat of environmental engineering’ with its 174 boreholes that provide natural geothermal energy, four ‘hybrid cooling towers’ and a ground source heat pump that will save enough energy to power 2,500 homes.

Add to that low-energy ventilation, high levels of insulation and a ‘saw-tooth’ roof design that minimises energy use by flooding the interior with natural daylight. AZ hopes the building will contribute to its Ambition Zero Carbon programme that commits to zero carbon emissions by 2025 and for its entire value chain to be carbon negative by 2030.

Following its recent approval of an injectable HIV-1 treatment from the National institute for Health and Care Excellence (NICE), GlaxoSmithKline (GSK) has high hopes in developing a cure for the virus.

UK pharmaceutical company GSK plans to begin human clinical trials of its potential cure for HIV in 2022. The company shared that it is aiming to develop a cure for the disease by 2030.

“Our ultimate goal is always a cure of HIV,” said Kimberly Smith, head of research and development at GSK’s HIV arm, ViiV Healthcare. She continued that she hoped the company would produce a cure “by 2030 if not sooner”.

The news follows approval from NICE in support of a long-acting injectable HIV-1 treatment, also developed by GSK, which ensures that treatment for HIV is only administered six days a year instead of daily pills.

GSK and Shionogi additionally entered into a deal in September 2021 in an attempt to extend the time between injections for HIV patients to three months or longer.

Current HIV treatment highly suppresses viral replication within the body, allowing the patient’s immune system to recover and regain the capacity to fight off infections and cancers.

The vast majority of people receiving HIV care in the UK in 2019 did so in England, according to the National Aids Trust (NAT).

Only a month after raising its 2021 outlook for both sales and profit growth, Novo Nordisk has announced that a new deal in China for its insulin portfolio will reduce global sales growth by 3%.

The company has been engaging in a tendering process with the Chinese government after the decision to bring all insulin products into the country’s centralised procurement programme in 2022.

Novo has announced that it was successful in getting all its insulin products onto the national formulary with the exception of Ryzodeg and Xultophy.

Ryzodeg contains a combination of long- acting insulin degludec – which Novo markets as Tresiba – and rapid-acting insulin aspart, which Novo sells as NovoRapid. Xultophy is a combination of insulin degludec and Novo’s glucagon-like peptide-1 (GLP-1) receptor agonist, liraglutide, which it markets as Victoza.

The assumption from Novo’s announcement is that Tresibo and NovoRapid, as well as NovoMix (insulin aspart/insulin aspart protamine) have been accepted by the Chinese government, albeit at a discount.

The value-based procurement (VBP) process in China will cover all insulins supplied in hospitals and is expected to be implemented in the first half of 2022.

After strong sales in the first nine months of the year, the company has raised its sales and 2021 growth forecasts to 12-15% but now estimates the ‘negative impact’ on global sales growth due to the deal will be around 3% in 2022.