Vertex Pharmaceuticals has directed their attention towards gene therapy and stem cell startups.
What is Gene therapy?
Gene therapy is the effective removal and replacement of a defective genome from an individual’s genetic makeup. The purpose is to treat and remove a genetic disorder or disease that affects a child’s developing stage.
As of yet, pundits and experts debate heavily of how Vertex intends to move forward in the industry. Keeping in mind the recent developments, which include regulatory approvals and multi-billion acquisitions, Vertex is expected to go on the offensive and work towards addressing major illnesses, such as sickle cell disease, beta-thalassemia, pain, myotonic dystrophy Type 1, Duchenne muscular dystrophy and alpha-1 antitrypsin deficiency.
However, no one’s entirely certain what the next logical step would be. This is primarily due to the ambiguities and high-risk investment involved. As of yet, there are only speculations. Professionals and analysts have, however, laid out the main attractions, such as the CRISPR. Nonetheless, it’s too soon to hold a firm belief in how Vertex might want to proceed with its acquisitions.
What lays ahead?
As of yet, Vertex has inspired confidence in its stakeholders. By bringing in about $3 billion in revenue last year, Vertex is projected to grow exponentially when a major triple-drug regimen ensures that patients of Cystic Fibrosis can acquire Vertex products for treatment.
Due to the prevalence of the similar disease, analysts often debate and conclude that Vertex pharmaceuticals will most likely continue on their research on Cystic Fibrosis, looking a way to serve 10% of the population afflicted by cystic fibrosis.
As focus on gene therapy grows, the upcoming CEO, Reshma Kewalramani, has promised a pipeline program that will address kidney diseases that are caused by a defect in the APOL1 gene. Due to the resources allocated, Vertex will primarily focus its attention on diseases that are due to single genetic mutations as opposed to a of a handful of defects.
Nonetheless, this does not inhibit Vertex’s reach and agenda for the near future. The acquisition of Semma promises treatment of Type 1 diabetes, which is linked with defects in multiple genes. Afflicting about 1.2 million people in the US alone, it’s fair to say that tackling this disease as soon as possible should be made a reality rather than speculation.
Gene therapy and stem-cell-based therapies may hold a promise. Vertex now races against time in successfully fixing single-gene mutations.