I’ve had a vague hope in gene therapy for many years, but have never really believed I would live to see it come to market as a real treatment.
I’ve told my family many times that “maybe gene therapy will be the fix”. Today is the first day I’ve actually believed those words.
No CRISPR/Cas9 was used in this study.
We'll have to wait and see if any of the patients develop hematologic malignancies (blood cancers).
I predict we'll see dozens of papers like this in the next few years. Sickle cell and thalessemias are low hanging fruit.
But there are other strategies in development to help some patients with autoimmune disease. This is one of my favorite papers in that vein: https://www.ncbi.nlm.nih.gov/pubmed/27365313
If you can't access it then shoot me an email at ron gejman at gmail dot com and I'll send you the pdf.
Anything changed in the DNA can have completely unforeseen consequences because genes and their products influence expression of other genes.
Which genes does each gene influence? Directly or indirectly? To what extent (strength of up- or downregulation)? Only under specific conditions? Only if 3 other genes are present and not regulated in a different manner?
I have done some computational gene interaction analysis by building regulatory interaction networks in Cytoscape using sequencing and expression data based on specific research .
My subjective impression is that messing with genes is little but an unpredictable endeavor at this point. The gamble may pay off but I don't think anyone can honestly tell you something about long-term effects in each individual.
(I'm not saying this as some kind of rrrgh-gubmint-bad thing. It's pretty normal for the government to have authority over what gambles are acceptable to offer in what circumstances, and which ones are exploitative and detrimental to even offer; this is the logic behind, for instance, regulations of actual gambling.)
If more common and widespread health problems are tackled with gene therapy and we wouldn't know the consequences long term (we really don't) then this aspect becomes far more problematic.
Like you, I would also take the gamble if it's a Hail Mary attempt at surviving or making a somewhat normal life possible. But that's not the point I was trying to tackle.
I'm hopeful that this is a panacea for this class of problem the likes of which we rarely see, but I will not be surprised if something tragic happens.
Where can I learn more about gene therapy as well as CRISPR/Cas9?
Specifically seminal papers?
Where can I learn more about the business aspect of it? Industry, projections, opportunities, etc.
Thanks in advance!
There are a number of "miracle cures" out there for genetic disorders that have been effectively discontinued or that have insanely high prices. I'm concerned that future articles about this breakthrough will be in that vein.
Hopefully not, but that's my fear.
Also, how are people aware of these small pharmaceutical companies and future breakthroughs like this without insider trading? Meaning, some people profited handsomely on this deal, how did they make it happen if not luck or insider trading?
The discovery of a new molecule or invention of a new method for treating disease is about 5-10% of the effort required to bring a drug to market. Yes, it's clearly not possible to bring a drug to market if it has not been invented, so it's essential to the process, but the difficulty shepherding this new thing through to commercial approval is fraught with difficulties. Universities and hospitals don't have the right talent to do this, nor do they have the resources or the risk tolerance so they sell some of the future gains to a company through licensing for money now. This is how almost all drugs are developed now-a-days, through licensing deals from the inventing parties. This brings the drug to market a lot faster, helping patients more quickly, and reduces the risk of development failure which would prevent patients from getting the drug.
On your second question, the biotech space is small and the number of investing analysts are also small in number. This means you can get a sense of the entire space by following a handful of people on twitter or by email. If you're in the business, you knew who's working on what. Biotech is unlike high-tech in that you need capital to do the work. Making an app can be done by a single person in their apartment. Making a drug requires millions of dollars, special lab equipment, the right people, being in a hub, etc.
In terms of insider trading, the SEC has strict rules on trading on non-public information and compartmentalizing this kind of information to a small group of people who have their trading accounts basically always watched by regulators. Every year a number of biotech employees go to jail for insider trading, so it clearly happens but the SEC is really vigilant on pursing it. The people who profited from this are likely the employees of the company and the investors. They didn't need insider trading as much as they needed to be in biotech, live in the Boston area, want a job working on gene therapy, and work for the company. It's like asking how did people make money off Facebook, or any other company that started-up and made it big.
Does anyone know if this can help GSD? My cousin has it and it’s an absolutely horrible disease, her childhood was robbed and her parents' lives upended https://en.m.wikipedia.org/wiki/Glycogen_storage_disease
This is manageable when you’re treating a white blood cell disease as you can live temporarily without these cells and they regenerate frequently.
But if all you need are some cells to produce more of an enzyme and can produce it in sufficient amounts to manage the existing cells producing the bad enzyme, you might be in luck.
This is poor phrasing. There are non X-linked SCIDs.