The AstraZeneca acquisition of LogicBio has brought an experienced team for rare diseases on board. It is a time-consuming and risky process to build such a team from scratch. The company’s team is working on multiple drugs for rare diseases. This acquisition makes it easier for them to deliver multiple drugs to their patients.
LogicBio’s MMA trial was put on hold
Following the news that the FDA had put a clinical hold on LogicBio’s MMA trial, shares of the gene therapy developer plunged 25%. The company has since announced that it will acquire LogicBio for $68 million. The deal is the largest ever for a U.S.-based biotech, and it is expected to create jobs in the biotech industry.
The FDA had put the MMA trial on hold after it identified two patients with a potentially serious adverse event (SAE). The two patients had received the investigational drug at a dose of 5 x 1013 vg/kg for the older age group. One of the patients’ SAE resolved after hospitalization, while the other patient experienced thrombotic microangiopathy after the treatment. The FDA said it would review the MMA trial on Jan. 21 and will allow the company to resume dosing the patients.
The company has two technology platforms that are focused on the delivery of genetic treatments. Its GeneRide platform harnesses the natural deoxyribonucleic acid (DNA) found in cells to deliver gene therapy. The company is also focused on adeno-associated virus (AAV) gene delivery technologies.
LogicBio’s “Gene Ride” platform
The deal also includes the transfer of all of LogicBio’s stock to Alexion for $68 million, representing a 600% premium over its closing share price of $0.27 on Friday. The company’s platform will help develop gene-editing treatments for rare diseases. The deal is expected to close within four to six weeks.
LogicBio’s inability to fully break down certain fats and proteins causes toxic byproducts to accumulate in the body
These byproducts can cause symptoms such as vomiting, weakness, coma, and death. However, the FDA has not approved LogicBio yet because it may not work in people with certain medical conditions.
The enzymes that are involved in the process are CACT, CPT, and ETFDH. These enzymes are involved in breaking down fatty acids. The CPT enzymes are responsible for converting fatty acids into acyl-CoAs. During this process, the fatty acid undergoes multiple cycles of beta oxidation, in which the fatty acid substrate is shortened by two carbons.
If LogicBio fails to fully break down certain fats and proteins, toxic byproducts are produced in the body. If the problem persists for a long time, the condition could lead to chronic health issues. Fortunately, most cases are caught early in childhood and can be easily managed.