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It may be a
challenge for 2019 to live up to the incredible discoveries and medical advancements made by the biotechnology industry in 2018. We saw the emergence of DNA and
gene-editing technologies that shook the industry, potentially changing the
course of future healthcare. More industrious research into curing cancer led
to even more innovative cancer treatments being brought to light. The first
ever cannabis-based drug was approved by the FDA, and we finally made headway
into tackling the antibiotic resistance crisis.
However, luckily,
2019 is shaping up to be just as impressive. Here are some examples of the most
revolutionary and promising biotech companies gearing up to make an impact this
year. There’s a good mix of familiar, dependable names as well as some smaller,
more niche biotechs brandishing new disruptive technologies and approaches to
addressing unmet medical need around the world.
Enterome
As chronicdiseases become more prevalent and an increasing threat to our health,
Enterome, a biotech based in France, focuses on developing drugs for microbiome
chronic diseases. In late 2018, Enterome partnered with Japanese biotech giant
Takeda to develop a treatment for Crohn’s disease, a chronic disease that
causes painful inflammation of the gastrointestinal tract.
The
serious condition is often linked with the inflammatory bacteria,
enterobacteria. The company is aiming to inhibit the bacteria by developing an alternative
to standard treatment, a non-antibiotic drug made up of small molecules,
peptides and proteins. This will not only provide a more effective treatment
for patients but also help in the fight against antibiotic resistance. The
drug, EB8018, is currently entering into late-stage phases of clinical trials.
Enterome’s
pipeline also features an out-of-the-box microbiome-derived vaccine that, if
successful, could stimulate the immune system to decimate cancer cells.
Alnylam
Based on
Nobel-prize winning RNA interference (RNAi) therapeutics, US biotech Alnylam gained
regulatory approval for the first ever RNAi treatment of its kind. The therapy
was developed to treat a very rare but life threatening disease known as hATTR,
and was effective in relieving and reducing symptoms and improving patients’
quality of life.
This is
just the first example of what can be achieved by exploring the power of RNAi
therapeutics, leading the way to an entirely new era of medicines that can treat
rare and devastating diseases. Alnylam has several late-stage (phases 2-3)
therapies in their pipeline, mostly to treat other genetic diseases but some
for cardio-metabolic diseases.
Many of
these have been awarded ‘breakthrough designation’ status, a regulatory process
used to fast-track drugs that treat serious conditions and have demonstrated superior
results to existing treatments early on.
Virion BioTherapeutics
Virion BioTherapeutics
is an innovative biotech that exists to tackle the 4th biggest
threat to our health: respiratory virus infections (RVIs) like the flu. Yearly influenza
vaccines are not always successful and there is currently no available
treatment for non-influenza viruses. To complicate matters, drug-resistant virus
strains are quickly emerging, making RVIs harder to treat and more deadly than
ever.
Virion is
developing a different treatment approach to RVIs that provides a high barrier
to resistance and prevents replication of the virus. It’s primary product VH244
uses ground-breaking viral interference methods to control viral replication
and to enhance a person’s immune response to the viruses. The treatment has
proved effective on a broad variety of RVIs and if successful could save
millions of lives from deadly, drug-resistant respiratory viruses.
Nouscom
Joining
the fight against cancer, Swiss-Italian biotech Nouscom is exploring the
potential of immuno-oncology. Their pipeline includes the first ever ‘off-the-shelf’
genetic vaccine to treat certain types of cancer, and another personalised
neoantigen genetic vaccine that targets tumours found in cancers like melanoma
and lung cancer. The vaccines use viral vectors from highly infectious diseases
like ebola and malaria to (safely) generate immune responses within humans,
which helps the body kill cancer cells.
The
personalised vaccine, NOUS-100-PV, is expected to enter the clinic in 2019 and
the first in-human clinical trial for the off-the-shelf vaccine, NOUS-209 is on
course to start at some point in early 2019.
Acceleron Pharma
Acceleron
Pharma is developing a range of innovative medicines in an effort to combat an area
of great unmet medical need: rare disease. The company’s discoveries are based
on the TGF-beta protein super family and hope to harness the body’s natural
capacity to repair itself.
Along with
biotechnology giant Celgene, a leading in the haematology space, the company is
on track to bring its leading product luspatercept to market. The therapy is currently
in late-phase clinical trials and is engineered to treat patients with rare and
serious blood disorders. Acceleron is also developing therapies to treat orphan/rare
neuromuscular and pulmonary (lung) conditions.
Pfizer
Although principally a pharmaceutical company, Pfizer is also
strong player in the biotech arena. The life science giant’s pipeline is brimming
with potential blockbuster therapies that are on track to gain regulatory
approval by 2020. In an attempt to provide more affordable and accessible
healthcare to patients worldwide, Pfizer is developing several biosimilars with
the intention of launching them within the next two years. Among them is one of
the best-selling biologic drugs, Humira.
Pfizer is also increasing its footprint in biotechnology by
investing an impressive $600 million in various cutting-edge companies that are
emerging with promising new compounds and technologies. The company is particularly
interested in furthering the progress of biotech companies working in neuroscience
realm, particularly neuro-degeneration, neuro-inflammation and neuro-metabolic
disorders.
Cleyad
Cleyad is
a Belgian biotech company that specialised in immune-oncology, one of the most
advanced ways of treating cancer. The company is working diligently to produce
an improved version of CAR-T cell technology, which has already taken the world
by storm with its innovative and effective approach to treating various cancers.
Cleyad’s
most exciting advancement is that it’s leading therapy has shown to be
effective in targeting solid tumours in patients who have not undergone
rigorous chemotherapy courses. If clinical results continue to show such
positive results, CYAD-01 could be the long-awaited alternative to harsh and
damaging regimes of chemotherapy and radiation.
Aelix
HIV continues
to be a prevalent and devastating disease that affects millions across the
globe. In 2018, there were a few HIV drugs approved which will bring new hope to those living with the disease,
and in 2019 the effort continues. Spanish biotech Aelix Therapeutics has made a
deal with Gilead, a leader in HIV research and development, to produce a
vaccine called AELIX-003.
This innovative
approach aims to not only control and supress a patient’s viral load, but to target
places where the virus is ‘hiding’ from the immune system. If successful, the
vaccine will be a welcome alternative to daily course of antiretroviral drugs,
and a more effective one. Aelix Therapeutics aims to eventually develop a cure
for HIV using this innovative vaccine platform.
Biogen
Our
understanding of the complexities of the brain is still developing and many
neurological diseases remain difficult to treat. One of the top 10 biotechnology
companies in the world, Biogen, is always on the radar when it comes to new innovative biotech therapies. The company has committed to developing its neurology and CNS
pipeline in 2019, with new therapies emerging for multiple sclerosis, Alzheimer’s
and Parkinson’s disease. It currently has 5 potential drugs for Alzheimer’s and
one for Parkinson’s.
Biogen is
collaborating with Skyhawk Therapeutics to develop medicines for multiple
sclerosis and spinal muscular atrophy (SMA) using its RNA platform. This technology
aims to tackle hard-to-target disease mechanisms that characterise many
neurodegenerative diseases.
CRISPR Therapeutics
CRISPR-Cas9,
a gene-editing technology, is one of the most ground-breaking discoveries in
recent years. The reason that the life science industry is so excited by this technology
is that it has unknown potential in treating an array of diseases. Swiss
biotech, CRISPR Therapeutics has begun a trial for the treatment of a blood
disorder called beta thalassemia using the technology. In 2019 the therapy will
be tested on humans to gauge its effectiveness.
CRISPR
Therapeutics pipeline also includes using the technology to develop immuno-oncology
therapies, regenerative medicines using stem cells and also to target genetically-defined
diseases through in-vivo.
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