The 12-Month Leap in Gene Editing Advancements

Did you know that across the globe, a staggering 320 million people grapple with the challenges of rare genetic diseases?

In fact, the impact is so profound that an astonishing 4,000 medical conditions are suspected to be a result of gene disorders. With figures like those, it was only a matter of time until the intricacies encoded within our DNA became a focal point for scientific exploration.

Although gene therapy is still a budding new research area in the expansive landscape of Life Sciences, 2023 marked a year of remarkable breakthroughs, spotlighting an intensified focus on unravelling the possibilities embedded within our genetic code.

Today, with a surge of Life Sciences businesses expanding their R&D activities to include gene editing capabilities, more researchers are hoping to harness the power of gene editing technology to tackle diseases that have long eluded conventional treatments.

So, as we comfortably settle into 2024, let’s delve into some of the most exciting gene editing breakthroughs that have unfolded over the last 12 months...

Nobel Prize-winning CRISPR technology has been turning heads in the world of R&D since its creation, showcasing unprecedented accuracy and efficiency in DNA editing.

In gene therapies specifically, the technology employs a guide RNA (gRNA) to pinpoint a specific gene’s DNA sequence. Once the target DNA is found, Cas9 – a key enzyme produced by the CRISPR system – acts like molecular scissors, binding to the DNA and cutting it, effectively deactivating the target gene.

With the potential to transform health outcomes for millions of patients worldwide, researchers have long explored CRISPR’s capabilities. Now, in the short 11 years since its initial discovery, the technology has achieved a significant milestone; it’s been granted its very first approval for gene therapy, Casgevy, that tackles sickle cell disease and beta thalassaemia.

On top of that, researchers focused on CRISPR have also made some significant strides in finding one-and-done treatments for conditions like Alzheimer’s, ATTR, HIV and ‘bad’ cholesterol.

Explore five exciting developments in gene editing science from the last quarter of 2023

Gene editing research, by its nature, involves a thorough and complex approval process. That means that the time it takes for these therapies to go to market can be much longer than other advancements within the Life Sciences space.

But that’s not to say that there haven’t been some noteworthy victories in the last 12 months! Let’s take a look at 5 other ground-breaking developments in gene editing R&D from 2023…

• A team of researchers from the Shenzhen Institute of Advanced Technology (SIAT) at the Chinese Academy of Sciences have pioneered a gene therapy strategy that’s aimed at selectively manipulating the affected circuitry involved in Parkinson’s disease. Their innovative approach, demonstrated in non-human primate studies, successfully mitigated the core motor symptoms of Parkinson’s disease. Tremor was eliminated, bradykinesia was significantly reduced, and motor skills were restored.Delve into the details of this promising research and how it could transform future Parkinson’s therapies
• A new biotech, AlveoGene, is embarking on a journey to develop inhaled gene therapies for rare respiratory disorders. To do so, the company has taken a significant step by exclusively licensing the InGenuiTy lentiviral delivery platform from the UK’s Gene Therapy Consortium. AlveoGene’s initial focus is on developing a targeted treatment for Alpha-1 Antitrypsin Deficiency (AATD), an inherited disease that leads to an increased risk of lung disease. With the aim of broadening their impact, the biotech plans to extend its efforts to explore potential treatments for other rare respiratory diseases, including lung surfactant deficiencies and idiopathic pulmonary fibrosis.Learn more about inhaled gene therapies and AlveoGene’s hope to address medical needs in respiratory health  
• Researchers at UC Berkeley and the Innovative Genomics Institute have joined forces to pioneer a therapy aimed at preventing the onset of asthma in children.Building on an identified link between the gut microbiome and childhood risk of asthma, the team is committed to treating the condition by targeting these specific microbes. They’ll work on precisely modifying the asthma-causing genes, ensuring accuracy while preserving the microbiome’s beneficial functions.Discover how researchers will use gene editing to prevent children from developing asthma
• In a strategic move, Cellectis has partnered with Primera Therapeutics to develop a gene-editing platform designed to tackle mitochondrial diseases. Leveraging Cellectis’s proprietary gene editing technologies, Primera is positioned to exclusively license up to five therapeutic candidates developed under the collaboration. The joint venture is dedicated to editing mutations in the mitochondrial DNA, with a specific focus on in vivo treatments targeting the root cause of diseases associated with mitochondria.Find out how this partnership is committed to advancing therapeutic solutions for mitochondrial disorders
• Cellectis has also recently teamed up with AstraZeneca, pooling their strengths to develop up to 10 cutting-edge cell and gene therapy candidates. The partnership will again leverage Cellectis’s proprietary gene editing technologies and advanced manufacturing capabilities while AstraZeneca will play the pivotal role of selecting up to 10 cell and gene therapy candidates from a pool of 25 genetic targets exclusively reserved for their collaborative efforts.Dive deeper into this collaboration and how it is set to elevate both companies’ gene therapy portfolios

This research area is undoubtedly one that will continue to innovate, defining itself as a pivotal sector within the ever-evolving Life Sciences ecosystem.

We can’t wait to see what further developments happen in the gene editing space throughout 2024 and beyond, providing patients with life-long conditions far better health outcomes.

What are your thoughts on gene editing therapies? Do you think they’ll grow to become a prominent field in the Life Sciences space? As always, we’d love to hear your thoughts!

Speak again soon!

The Meet Team