Emerging technologies in biotech

Ditte Funding, Natasha Cowan|25th September 2017

In 2016, the FDA’s Centre for Drug Evaluation and Research approved 15 new molecular entities (NMEs) – innovative products that serve unmet medical needs or make significant strides in advancing patient care and public health.

While the number of NMEs in 2016 dropped by more than 50%, things are looking up for 2017 with FDA Novel Drug approvals already exceeding 2016 figures. In particular, we believe there are a number of transformational technologies emerging from biotech at the moment with the potential to change the face of healthcare.

Here we identify four complex platforms to watch, and reveal how biotech companies across the world have championed these innovations, overcoming significant challenges to address patient needs and advance medical care.

CAR-T

We have written extensively at Blue Latitude Health on the impact of immunotherapies in oncology (IO), now the next generation of IO treatments are here and CAR-T is taking centre stage.

The treatment fights cancer by engineering T-cells, which are collected from the patient’s blood, to both recognise and attack tumour cells. The CAR-T cells are then infused into the patient where they multiply to find and kill cancer cells.

It has not been smooth sailing for CAR-T. During the past few years, severe side effects have been reported, such as the death of three patients as a result of cerebral edema in Juno’s JCAR015 phase II trial. These have resulted in strict regulation and scepticism around the safety of this technology. The FDA suspended Juno’s trial and the company’s stock price plummeted by almost 30% as a result of the fatalities.

Recently things have been looking more positive for CAR-T and we predict 2017 to be a momentous year for this technology. Early trials by Novartis studied paediatric patients who had run out of treatment options for advanced acute lymphoblastic leukaemia. Astoundingly, within three months 52 out of 63 patients achieved complete remission, or complete remission with incomplete blood count recovery. However, many of the patients experienced severe cytokine release syndrome as a side effect.

The FDA recently hosted a 10-member Oncologic Drugs Advisory Committee (ODAC) to determine whether the benefits of CAR-T therapy outweigh the risks when treating paediatric leukaemia. The response was unanimously positive, with panel member Timothy Cripe of Nationwide Children’s Hospital in Columbus, Ohio, describing the treatment as “the most exciting thing I’ve seen in my lifetime.”

However, a major challenge for companies such as Novartis, whose drugs are nearing marketing approval, is ensuring they can price the therapy at an acceptable rate for healthcare systems, while still earning a profit. The manufacturing process is complex and costly. Each treatment is individualised and the patient’s blood is shipped to a lab where it is modified, manufactured and then shipped back to the patient.

Critics are describing CAR-T as the beginning of a new era in oncology. On August 30th 2017, Novartis’ Kymriah was approved in a move the agency itself described as historic. Now, it is only a matter of time until patient’s begin to see the benefits.

CRISPR

This cutting-edge, gene-editing technique is causing a stir because not only is it a major discovery in the scientific sphere, it’s accurate and relatively cheap to use. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.

The technology works by replacing mutations and reverting genes back to their normal sequence. The DNA editing technique is based on the way bacteria defend themselves from viruses. Scientists behind CRISPR-Cas9 took this technique and transformed it into a gene-editing tool. The bacterial ribonucleic acid (RNA) system is modified to tell the enzyme Cas-9 where to look for certain DNA. When the DNA is found, the CRISPR system binds to it and slices it, shutting down that specific gene.

Plans for CRISPR have ranged from creating malaria-proof mosquitos and ending peanut allergies, to bringing back the woolly mammoth and designing crops that can withstand climate change.

In July, the ‘MIT Technology Review’ reported that a team of scientists in Portland, Oregon led by Shoukhrat Mitalipov of Oregon Health and Science University, took the pioneering step of using CRISPR to edit the genes in a human embryo in the US.

The controversial treatment fertilised donated human eggs with sperm known to carry disease-related mutations. Mitalipov and his team corrected the mutation using CRISPR and let the embryos develop for a few days before destroying them. Remarkably, tests show the desired genetic changes took place with few errors.

While the UK and US have given the green light to the use of the CRISPR-Cas9 gene editing technique, the practice remains highly controversial, and the approval process for the technology is extremely strict. However, this has not slowed enthusiasm from the scientific community.

Vertex and Bayer recently partnered with CRISPR Therapeutics to get in on this cuttingedge technology and broaden the range of indications, leading us to predict this is only the beginning of CRISPR’s story.

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