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Div­ing deep in­to AMD, Yale re­searchers pin­point cell types drug de­vel­op­ers should pay spe­cial at­ten­tion to

The wild successes of drugs like Eylea and Lucentis sometimes overshadow the fact that there still isn’t a cure for age-related macular degeneration, the leading cause of blindness among the elderly, especially the earlier-stage disease known as dry AMD. While around three dozen genes tied to the disease have been identified — giving rise to experimental gene therapies — some scientists believe a closer look is needed to figure out where exactly these genetic alterations are taking place.

Can CBD tem­per Parkin­son's-re­lat­ed psy­chosis? UK re­searchers will look for an­swers in PhII study

Cannabidiol (CBD), the cannabinoid compound found in the cannabis plant, has been tried and tested in patients with rare forms of epilepsy, inspiring the approval of GW Pharma’s $GWPH landmark plant-derived Epidiolex. Its resurgence in research — and its ubiquitous presence in oils, creams, and gummy bears — is on the basis that it is not addictive, like its intoxicating cousin THC, and retains the therapeutic potential for a plant that was once touted as a cure-all in India. Researchers at King’s College London have been studying the effect of CBD on psychosis, and on Monday signaled they are prepping to begin a large-scale Phase II trial in patients with Parkinson’s-related psychosis, characterized by hallucinations and delusions.

William Kaelin. HHMI

What does No­bel-qual­i­ty re­search look like in ac­tion? Re­searchers re­count the Bill Kaelin ex­per­i­ment

There’s a question banded about at Tango Therapeutics, the biotech William Kaelin co-founded a few years back. It’s the adult scientist equivalent of a math teacher asking if you checked your work, subtracted back the sums and multiplied the quotients: Have you done the Bill Kaelin experiment?

“He holds it almost as a principle,” Tango CSO Alan Huang told Endpoints News. “And he won’t let go.”

Gregg Semenza
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William Kaelin, Peter Radcliffe, Gregg Semenza

Trio wins No­bel Prize for Med­i­cine for 'fun­da­men­tal' dis­cov­ery

The Nobel Prize for Medicine was awarded to William Kaelin, Peter Ratcliffe and Gregg Semenza for a discovery on how cells respond to oxygen that could have a wide-ranging impact on treatments for anemia and cancer, among other ailments.

The researchers earned Nobel status for their identification of the HIF, or hypoxia-inducing factor, protein that controls how the body responds and adapts to oxygen levels, and a host of related discoveries. The findings have yet to produce actionable therapies, but HIF has been shown to impact tumor production — helping cancer cells compensate for an oxygen-low environment — and red blood cells.

Brendan Frey. Deep Genomics

Cana­di­an star re­searcher Bren­dan Frey bran­dish­es Deep Ge­nomic­s' first AI-dis­cov­ered drug

A startup harnessing AI in drug discovery has put forward its first therapeutic candidate — and it may not be who you’re thinking.

To be sure, Deep Genomics still has a ways to go in preliminary animal work to ensure safety and non-human primate as well as biodistribution before they can move into clinical testing. But the fact that its AI system was able to go from target identification to declaring a winner in 18 months, said CEO Brendan Frey, is a game-changer.

Frey founded Deep Genomics in Toronto in 2014, a couple years after more well-known players such as Atomwise, BenevolentAI and Recursion popped up, based on a decade of research on how AI can help scientists understand genetic diseases. While other platforms have tended to zero in on one part of the puzzle — Atomwise is focused on accelerating small molecule screening, Recursion relies on cell imaging, and insilico uses AI to modify existing drugs — its tech “does the whole thing.”

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Biotechnopark in Koltsovo, Novosibirsk Oblast. Shutterstock

Ex­plo­sion, fire rip Russ­ian re­search fa­cil­i­ty hous­ing small­pox, Ebo­la

An explosion started a fire at a Russian research center that houses smallpox and Ebola, blowing out glass windows and inflicting third-degree burns on one worker, although state-run media said that there is no danger to the general population.

The State Research Center of Virology and Biotechnology, also known as the Vector Institute, is one of only two places in the world that still houses the smallpox virus, after the WHO declared the disease eradicated in 1980. The other is — somewhat controversially — the US Centers for Disease Control (CDC) in Atlanta.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

How does TDP-43 bog up the brains of ALS, FTLD pa­tients? AC Im­mune en­lists Penn sci­en­tists to find out

AC Immune, the Swiss biotech known for its tau approach to Alzheimer’s, is shining light on another misfolding protein believed to be responsible for other neurodegenerative diseases in a new research pact with the University of Pennsylvania.

The transactive response (TAR) DNA binding protein, or TDP-43 in short, is found in most human tissues but its misfolded form was identified in 2006 as a common pathologic substance linking frontotemporal lobar degeneration (FTLD) and amyotrophic lateral sclerosis (ALS). Researchers have since suggested that when in the wrong shape, the protein activates the “cell autophagy gene” ATG7, which prompts neurons to decompose.

Bay­er’s $240M up­front for Blue­Rock ac­qui­si­tion puts them in the high rollers club for pre­clin­i­cal biotech buy­outs

Bayer’s deal today marks a major, top-10 acquisition for a preclinical biotech, but it’s also significantly different than the average big-buck pacts in the field, which typically revolve around a single drug or target. Bayer wants to build a whole new pipeline covering a variety of disease fields.

The pharma giant’s $600 million gamble — $240 million in cash upfront with $360 million for development goals — ranks as the 6th largest money-down deal on the top 20 list of preclinical M&A deals, as gathered by DealForma’s Chris Dokomajilar.

The list is topped by Bristol-Myers Squibb’s misguided $800 million upfront for Flexus’ IDO program — later scrapped. Tekmira — now Arbutus — followed with a motherlode of dollars for OnCore’s hep B effort. That stands out as an unusual play for a biotech. The top 15 players dominate the preclinical M&A game when it comes to large upfronts.

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Adrian Hayday. Crick

Adri­an Hay­day re­futes chal­lenge on in­flu­en­tial au­toan­ti­body pa­per by Karolin­s­ka sci­en­tists

Back in 2016, Adrian Hayday and his colleagues at King’s College London — together with some collaborators in Germany and Estonia — made a splash in the immunology world with a paper, published in Cell, suggesting that patients with a particular genetic defect produce more autoantibodies than previously known. These self-generated antibodies, they proposed, offered therapeutic potential for autoimmune diseases, most prominently Type 1 diabetes.