Early Success for Hemophilia Gene Therapy

Diseases that are genetically inherited are a little tricky to deal with. Since they don’t manifest themselves early on and have the potential to absolutely skip a generation, complications are bound to arise. Furthermore, more complications hinder progress if such diseases have a high-risk factor.

On the top shelf of such diseases, hemophilia is arguably the worst one. Hemophilia results in the recipient not being able to produce any blood clots. Since blood clotting is necessary for the prevention of bleeding, the victim is always under risk from any sort of threat, whether it is external or internal.

What causes hemophilia?

Hemophilia is mainly caused by irregularities and mutation of the Factor VIII gene. This is arguably the most important factor for blood clotting. In patients diagnosed with hemophilia, this gene is missing. Furthermore, hemophilia is passed on from one generation to the other, rather than being contagious or airborne.

Treating Hemophilia

Sangamo Therapeutics has been conducting experiments and trial to discern the root causes and how to go about treating them. Due to the high-risk factor, not many patients are a part of this meticulous process. Patients that were the high dosage of medicinal drugs were repeatedly tested and kept track of and showed a consistent level of Factor VIII.

The experiments have been successful as far as the gene concentration is concerned. The major player, Factor VIII, manifested itself perfectly in its patients and functioned normally. However, some patients reported some minor side-effects and discomfort, where one patient claimed low blood pressure and fever about six hours after dosage.

The dosage also referred to as SB-525, is arguably one of the breakthroughs that the biotechnological company has made in a while. Showing immense promise, this is arguably said to serve as a pivotal role in the development of this novel gene therapy technique which is bound to tackle rival hemophilia gene therapies.

What lay ahead?

As of yet, no statement can be issued without certainty. However, two of the main players in this industry are Sangamo therapeutic and Bio Marin Pharmaceutical. Confident of their ability and building off of results that the others publish, it is argued that major development and progress is expected to materialize in about the next 18 months. Till then, the main run-through of the trial would have been run.

However, as far as goodwill is concerned, it is of tantamount importance to understand and realize the fact that both of the companies are competing to tap into a unique market. Since the issue of definite hemophilia gene therapy hasn’t been completely addressed yet, the first individual to go to the open market wins. This gives both of these pioneers enough incentive to accelerate their research methodologies so that substantial information is obtained.

Another player in this scientific rat race is Spark Therapeutics, who claims that their therapy would include a very low dosage which would have the same effect. Claiming to hold a conference in mid-2019, Spark ensures that their product will be unparalleled and might perhaps be the final solution.

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