060 | Intellipharmaceutics’ face lawsuit over tamper resistant opioid, IDT tackles CRISPR off-targets

The ups and down of an Ontario-based pharma company seeking to develop a solution for the world’s deadly opioid epidemic. A custom DNA nucleotide maker locks in on the problem of CRISP Cas9 off-targets I’m Nestor Arellano. And these are the biotech news we’ll talk about today on the Biotechnology Focus podcast +++ Intellipharmaceutics International of Etobicoke, Ont. was decidedly upbeat when it announced a couple of weeks ago the upcoming review by the FDA of its tamper resistant opioid medication called Rexista. Rexista was product squarely aimed at an unmet need. In recent years, Opioid addiction has become a major problem and growing cause of drug overdose deaths in Canada and the United States. At least 2,458 people were reported to have died in Canada last year due to opioid-related overdose. INtellipharma’s product appeared to promise to turn the tide. Rexista was being positioned as an abuse and alcohol-deterrent . It was a controlled-release oral formulation of oxycodone hydrochloride. A Bioequivalet of Purdue Pharma’s OxyContin. Among other things, Rexista contains a blue dye which would mark those who tamper with or crush the tablet. It also contains a nasal irritant designed to keep the drug from being easily inhaled with the application of heat or an open flame. Wow, the FDA review must have looked like a slam dunk. But a few days later, reports came out that a Los Angeles law firm to investigate Intellipharmaceutics and its company’s officers for possible violation of federal security laws. The complaint, filed by a certain Shawn Shanawaz against Intellipharmaceuticas and two of its executives, alleges that the company and its officers violated Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 by making allegedly false and misleading statements or failed to disclose certain information regarding Rexista. Then another law firm law firm Glancy Prongay & Murray LLP (GPM) of L.A. issued a press release saying that it was looking into reports of alleged misconduct by Intellipharmaceutical executives. The press release indicates that the investigation may have something to do with share prices of Intellipharmaceutical. According to GPM the FDA committees voted that Rexista “had not demonstrated properties that can be expected to deter abuse” by the intravenous means and that there was not enough data to support the company’s claims of the product’s abilities to deter abuse by this means. “On this news, the Company’s share price fell $1.13 per share, or 45.3% on July 27, 2017, thereby injuring investors,” GPM said. The law firm was inviting eople who have purchased Intellipharmaceutics or anyone that may have information or questions about the claims to contact GPM. The company’s CEO had earlier said “We are very pleased with the progress made towards our goal of securing FDA approval of our RexistaTM NDA candidate.” A few days later, Intellipharmaceutics issued a statement saying “its management intends to vigorously defend against the allegations set forth in the complaint.” ++++ The ability to modify the gene of an organism through CRISPR genome editing holds the promise of curing diseases such as cancer and leukemia. However, there are also growing concerns that the innovative gene editing technology could alter regions of the genome which researchers are not targeting. A California-based custom manufacturer of DNA and RNA oligo nucleo tides believes it can lick this problem. The company launched a Cas9 enzyme variant which it said can drastically reduce off-target effects in CRISPR genome editing. Integrated DNA Technologies (IDT) said the Alt-R S.p. HiFi Cas9 Nuclease 3NLS enzyme Ok that’s a mouthful. Let’s just call it variant enzyme. The variant enzyme is a recombinant S Pyogenes Cas9 mutant The company said that when used in gene editing it improves specificity while maintaining a high editing efficiency similar to wild-type Cas9. IN short the variant enzyme is able to cut down off target effects. Why is this important? CRISPR Cas9 editing is not as accurate as we would want it to be. There have been warning from experts that it is possible to cause the editing an unintended gene. For example, genome editing could inadvertently disable a tumour-suppressor gene or activate a cancer-causing gene. Or, consider the possibility of an off-target effect where two different chromosomes are joined in a phenomenon called translocation. Translocation is the cause of chronic myeloid leukemia and other conditions. Yeah. That’s bad news. But IDT’s variant enzyme seems to be right on target. “We performed an unbiased evaluation of several versions of high fidelity Cas9 enzyme in primary human stem cells,” said Dr. Matt Porteus from the Stanford University’s Division of Stem Cell Transplantation and Regenerative Medicine. “We have been very impressed with the characteristics of this new IDT enzyme,” he said. He said that unlike other versions, the variant enzyme consistently achieved high on-target editing activity while having low off-target activity. Porteus was so happy with the results he even said his team intends to use the variant enzyme in future experiments on developing genome editing therapies. If you’re interested to learn more about this story Head over to our site Biotechnologyfocus.ca and click on the story: IDT launches Cas9 enzyme which cuts down CRISPR off-targets +++ Ok folks, that all the time we have for today. I hope you enjoyed the stories we just discussed. This is your host Nestor Arellano Inviting you to joins us again next week for more biotech news and views