Bioshots

Your bioshots for the week:

1. WAVE Life Sciences: FDA approves orphan drug designation for its lead candidate against Huntington’s disease

WAVE produces stereopure nucleic acid drugs (antisense oligos, ASOs) which reportedly improves their pharmacology. ASOs are commonly modified with phosphorothioate that improves their stability in vivo but this also introduces a new chiral center. CEO Paul Bolno (former Head of Neuroscience/Asia Business Development at GSK) states that an oligo with 20 nucleic acids has 19 chiral centers, providing a massive number of permutations that often result in varying pharmaceutical properties.

WAVE’s lead candidate called WVE-120101 targets rs362307, a Single Nucleotide Polymorphism (SNP) associated with the mutant huntingtin (HTT) gene, enabling a selective therapy that would not affect normal HTT protein levels. A competitor, Ionis Pharmaceuticals (formerly known as Isis Pharmaceuticals), has another antisense RNA oligo therapy currently in PI/PIIa clinical trial in collaboration with Roche, but this candidate targets both wildtype and mutant forms of HTT.

2. CRISPR-editing enters human trials

Scientists at University of Pennsylvania have been granted approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes of Health to perform the first human clinical trial using CRISPR-modified T cells. And Tech mogul, Sean Parker, will be funding the trial with his newly set up $250 million Parker Institute for Cancer Immunotherapy fund. Three mutations will be introduced on two genes in patient-harvested T cells, two to give cells an effective receptor that recognizes a protein (NY-ESO-1) only expressed on cancer cells. Another against PD-1, which cancer cells use to hide from immune surveillance. The same team had recently done a study looking at whether CRISPR would affect off-target genes, and their findings appeared to be sufficiently assuring. STAT news highlighted several concerns with the trial, including the monetary incentives that researchers are under, which historically have led to more successful trial results often related to researcher bias. Approval from the FDA still has to be obtained before the trial can proceed.

3. Non-coding RNAs, not so non-coding

Scientists have found many micropeptides are translated from non-coding RNAs (see here for a previous post on ncRNAs). These micropeptides were previously overlooked as algorithms were configured to find open reading frames (ORFs) only longer than 300 bases (100 amino acids), in order to filter out the numerous shorter ORFs that do not code for proteins. Techniques such as ribosomal footprinting and next generation sequencing however have enabled the detection of these micropeptides which can then be evaluated for biologically relevant functions. Several have already been found to play important roles in immunogenecity, embryonic development and muscle contractility.

4. Brexit – Impact on Science

Chaos strikes the world as the UK referendum voting results called for UK to leave the European Union. This does not bode well for research in UK as the EU funds a large proportion of research in the form of research grants or PhD/Post-doc fellowships. From 2007-2013, EU funding support in the UK was approximately $9.8 billion as opposed to $6 billion granted by the UK government.  London-based European Medicines Agency (EMA)—the EU version of the US Food and Drug Administration (FDA)—will likely also relocate as a result of Brexit. See here for a list of quotes from prominent figures and their views on Brexit and impact on biotech/research.

5. Amgen and UCB Pharma report positive PIII results of osteoporosis drug, romosozumab

At Endocrine Society’s annual meeting in Boston, Amgen/UCB released results from their P3 trials of romosozumab, a humanized monocloal antibody that inhibits sclerostin, increasing osteoblast activity and bone building. The study found improvement in hip bone mass density over 12 months compared to placebo and was accompanied by lower incidence of clinical fractures in post-menopausal women with osteoporosis.

6. Women’s contraceptive choices over the years and other informative graphics

It appears IUD’s are making a comeback along with implants and patches. Women are also giving birth later (no surprise) and interestingly no new antibiotics have been discovered ever since 1984!

7. China’s research boost

It appears China’s anti-misconduct campaign is working as number of misconduct allegations fall. The National Natural Science Foundation of China (NSFC) is working hard to raise the bar on quality and have come up with a plan from 2016-2020 that includes establishing a deep underground Earth-physics lab near Sichuan to detect dark matter, and launching 24 scientific satellites over the next 5 years.

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One thought on “Bioshots

  1. Pingback: CRISPR trial in humans – a look at checkpoint inhibitors | Bioshots

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