#dnai

https://www.reuters.com/investigations/ai-enters-operating-room-reports-arise-botched-surgeries-misidentified-body-2026-02-09/

MORE OBAKE & KEI HEAD CANON TIME - cuz nok's just a liiiiittle bit obsessed -, but focused less on relationship stuff, SO HERE GOES:

- Obake has been rather sickly since he was a kid. The moment you hear that a flu starts going around, you can expect to find Bob lying in his bed with a fever right away. His fevers usually get pretty high when he's ill & he hallucinates a lot. He'll tell you that that's a lie, but Kei will tell you nothing but the pathetic TRUTH.

- He really has no functioning appetite & has to be reminded to eat & sleep, otherwise he'd just keep on going until he dies lol

- Because of his - ah, shall we say "line of work?" - business, he's become completely nocturnal. Kei tries to force him to have a more normal sleep schedule, but considering he already sleeps so little, it's a disaster doing so.

- Obake keeps in touch with his parent's very infrequently & always joins them to celebrate Christmas, much to his annoyance. His parent's believe that their son owns his own small technology company & is doing ok for himself. His younger, overly successful brother is the only person in his family that thinks otherwise & is more aware than he should be of Bob's dirty dealings...

- Kei never mentions it until after Liv starts sending out mutants across San Fransokyo, but she has a twin sister named Ai.

- When their parents split, whereas Kei became more obnoxious & loud to gain attention, her sister withdrew, becoming quiet & fixated on books & science. Ai works on creating the perfect hybrid creatures & her biggest idea is to create man's true best friend, using genes of cats, dogs & foxes. She has one functioning example of her work named Jazzy-Lu, who is the most helpful & loyal creature ever! Ai is rather grey morally aligned & she enjoys causing chaos, even if it is at her own sister's expense.

- Obake & Ai are alike in many ways, but they both really don't care for one another lol

- Kei goes to her sister for help tho because Liv once stole research from Ai for one of her own pet projects & ever since then, the two have had a firm rivalry. Ai would love nothing more than to ruin Liv's fun!

- Backstory time! Both twins grew up in New York & only moved to San Fransokyo after their great aunt died, who left behind her odd mansion to the girls, as they were pretty much expelled by the rest of their family & their aunt wanted to give them some place where they would belong after her passing. Their aunt was a dabbler in plant based gene splicing & operated a successful vineyard that produces it's own labels & everything.

- The sisters are barely scraping by financially because the vineyard operates separate from them & only the mansion was gifted to them, but they're both grateful at that! Kei chose to move into the city for work & later became DJ Parasite!!... & Ai stayed behind to continue her studies.

- Shortly after she moved in, Ai accidentally stumbled upon a secret lab in the old green house & discovered a key that their aunt left behind for them as one last surprise for the girls. Ai wears the key around her neck & still has no idea what the key goes to.

ProNAi Therapeutics Inc (NASDAQ:DNAI) just reported that it is set to deliver interim results from an ongoing phase II in its lead oncology candidate at the 52nd Annual Meeting of the American Society of Clinical Oncology (ASCO). The event is scheduled to take place on the 6th of next month, and if market interest in the release date announcement is anything to go by, it could have a considerable impact on the company’s near term valuation. With this in mind, here’s a look at what’s set to be presented, and what to keep an eye on as indicative of bullish or bearish response.

So, the drug. A wave of oncology drugs has hit the development space over the last half decade, and many are simply iterations of one another in a particular family of molecule. ProNAi’s candidate, however, is a little different. It’s called PNT2258, and is what the company refers to as a DNAi. DNAi is another word for a single stranded DAN oligonucleotide (think of this as a type of building block of DNA) surrounded by a lipid outer coating. A team at Wayne State University figured out that these oligonucleotides can interact with certain oncogenes (which are the mutated genes that cause cancer) and affect their behavior. ProNAi picked up the tech from Wayne State, and developed PNT228, which specifically targets an oncogene called BCL2. BCL2 is an oncogene that impacts apoptosis. Apoptosis is the process through which cells induce their own death. In cancer cells, apoptosis isn’t as efficient as it should be, and cells that should die, don’t. Through the introduction of PNT2258, ProNAi is hoping it can reverse the the apoptosis inhibition that BCL2 induces, and in turn kill cancer cells. Specifically, and as under investigation in the trial in question, relapsed or refractory diffuse large B-cell lymphoma cells.

So what are we looking for in the upcoming trial data?

The trial in question is called Wolverine DLBCL, and it is set up to investigate both efficacy and safety concurrently. Each patient will receive the drug intravenously, 120mg/m2, on the first five days of a 21-day cycle. The administration period lasts for six months total. The primary endpoint is overall response rate, and while the measurement point for this overall response is not explicitly clear, in all likelihood the ORR in this instance refers to tumor size. So, we are looking for a marked reduction in tumor size (in B cell lymphoma, these tumors are generally in the lymph nodes) as an indication that the drug is effective in treating this indication. A host of secondary endpoints are in play, including time to response, which just means how long did it take for the tumor in question to start reducing in size, and overall survival. OS ranges from 20-50% in this sort of cancer across a five-year period. The measurement will initially address a 24-month period, but we should get some indication of it comparable efficacy (versus current SOC) even at this early stage.

What’s the upside on approval for the drug? If ProNAi can carry PNT2258 through to commercialization, it will gain access to a multi billion-dollar market. A recent analysis suggested that treatment market is set to rise slowly in value from $4.38 billion in 2014 to reach $5.45 billion by 2024. The company can’t expect to get full penetration, of course, but anywhere in the low to mid teens would still offer up a considerable premium on its current market capitalization, which came in at al little over $150 million at last count.

With this in mind, if the data comes out as indicative of the drug’s efficacy, we can expect some immediate upside in ProNAi. It’s the company’s lead candidate, and the technology that underpins its MOA is the underlying technology behind much of its wider clinical and preclinical pipeline. As such, the downside could be just as severe as the upside could be rewarding, if the data suggests the technology doesn’t work in a clinical setting. Risky, but one to keep an eye on if phase II interim comes out positive. The poster will be released at the company’s website premarket on June 6, and the presentation will take place the same day.

The Food and Drug Administration approved 44 drugs in 2015. Of those 44, 17 were orphan drugs, defined as addressing less than 200,000 people in the United States. That’s almost 40% of all drug approvals last year. In 2014, approvals were even more skewed in favor of orphans. Of the 41 drugs that gained FDA approval in 2014, 17 again were orphans. That’s over 42%. In the last two years then, 40% of all new approved drugs have been orphans, definitely outsized representation given that orphan diseases are by definition rare.

Orphan designations keep coming in, as companies seek them for expedited regulatory approval and longer patent exclusivity. 2016 has already seen its share of headlining orphan designations for both Big Pharma and small alike. Statistically, the chances are much better at getting regulatory approval with orphan designation than without, so the trend should continue. Here are four companies that were recently granted orphan drug status for some high profile clinical candidates.

AstraZeneca

AstraZeneca PLC (NYSE:AZN) has received two recent orphan designations from the FDA. One came in late February from European regulators for acalabrutinib, targeting lymphoma and leukemia. AstraZeneca believes that sales could hit $5 billion a year if the drug gets approval. AstraZeneca bought the rights to the drug for $4 billion from privately held Acerta Pharma, and so far results show a 95% response rate, with acalabrutinib being trialed in 20 different indications concurrently.

More recently, AstraZeneca was able to get orphan designation for its monoclonal antibody MEDI-551, developed by MedImmune LLC. The antibody treats an autoimmune condition called neuromyelitis optica, or Devic’s disease. It is similar to multiple sclerosis which attacks the myelin sheath around motor neurons, but in many ways worse. Devic’s disease attacks the myelin sheath around the optic nerve and spinal cord, eventually causing blindness and paralysis. A Phase II/III trial is currently underway with results scheduled around January 2018.

ProNAi

ProNAi Therapeutics Inc. (NASDAQ:DNAI) is an interesting one, a relatively new company having just been added to the Nasdaq Biotechnology Index in December. The stock has not been doing well since its July 2015 IPO, down almost 80%, but new orphan designation for its lead candidate PNT2258 could help reverse fortunes. PNT 2258 targets diffuse large B-cell lymphoma, or DLBCL, and is currently undergoing a Phase II 61 patient study scheduled to complete in October.

PNT2258 is unique in that it is a gene therapy that attempts to alter oncogenes, or cancer causing genes, by fitting them with oligonucleotides, which are short single-stranded sections of DNA code. The theory is to bind the cancer genes with the code, which alters the instructions to the cancer cells, allowing them to die. It essentially inhibits bad cancer-causing DNA, hence the company’s symbol DNAI, or DNA inhibitor.

In previous clinical trials, 100% of patients with DLBCL responded to the drug, with 2 complete responses. The trial was small though with only 13 patients, 4 of whom had DLBCL. Still quite promising though.

DelMar

DelMar Pharmaceuticals Inc. (OTCMKTS:DMPI) was awarded a second orphan designation for its brain cancer drug VAL-083 last week, March 15. It now has orphan status for two brain cancer indications, glioblastoma and medulloblastoma. VAL-083 is a chemotherapeutic that has been known about for decades for its anticancer activity, but it was never fully developed. It is different from the current glioblastoma standard of care chemotherapy temozolomide because its activity against cancer cells is not affected by MGMT, a DNA repair enzyme that helps brain tumors resist temozolomide.

This makes it a potentially better glioblastoma drug than the current standard of care. The drug has been extensively tested in early stage trials in the past, and results show a clinically meaningful survival benefit over other salvage therapies for those resistant to temozolomide. A Phase II trial is fully enrolled with an expansion cohort at the maximum tolerable dose, and a Phase III registrational trial is planned for later this year. DelMar has recently stated its intention to develop VAL-083 on its own rather than seeking to partner and license out, picking glioblastoma as a target because its small circumscribed patient base and opinion leaders lends itself to easier market penetration.

On top of all this, VAL-083 is already approved in China for the treatment of blood and lung cancers. Together with an orphan designation, chances look decent for eventual approval.

Bellicum

Another intriguing orphan designation winner this year, Bellicum Pharmaceuticals Inc. (NASDAQ:BLCM) was granted orphan status for its own lead candidate BPX-501. This drug is more a treatment for cancer treatment rather than a cancer treatment itself, with big potential. The issue is finding matches for patients with blood disorders, both cancer and non-cancer who need full hematopoietic transplants.

A full match often succeeds for hematopoietic transplants, but is extremely hard to find. Partial matches are easy to find from immediate family members, but risk the deadly complication of graft vs host disease, or GvHD, which happens when the T-cells in the graft blood attack the host body. In order to lower the chances of GvHD for partial match transplants, the T-cells are first removed from the blood, but this introduces immunodeficiency similar to AIDS, and is itself life-threatening. Because of this conundrum, partial match transplants are often avoided altogether as too dangerous.

BPX-501 is designed to add in genetically modified T-cells with a safety switch post transplant, without increasing the risk of GvHD. If it works, it would enable people who cannot find a perfect match to use imperfect matches when needing a hematopoietic stem cell transplant. This vastly changes the survival picture for patients with blood disorders of all types, as it is relatively easy to find partial matches.

In Phase I/II results reported for non-cancer patients (cancer is being reported separately), BPX-501 resulted in zero transplant related mortalities among 37 patients who underwent partial match transplants, with all 7 cases of low-grade GvHD resolving on their own. Patients treated with BPX-501 achieved acceptable T-cell counts 40 days faster than control. So far so good, and the new orphan status should help advance it toward a registrational trial.

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marg por favor

dijiste que te ibas a bañar hace 15 minutos? lol…….