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.

In a new pa­per pub­lished in Na­ture Com­mu­ni­ca­tions, a group of Yale re­searchers re­port­ed that macroglia, mi­croglia, vas­cu­lar cells, and cone pho­tore­cep­tors are the main cell types as­so­ci­at­ed with the risk of AMD.

Through per­form­ing par­al­lel sin­gle-cell RNA se­quenc­ing on 20,901 cells re­trieved from post­mortem hu­man reti­nas, they were able to con­struct a sin­gle-cell tran­scrip­tom­ic at­las of the eye tis­sue.

How is this sig­nif­i­cant? From the pa­per:

It was pre­vi­ous­ly thought that AMD was due to pathol­o­gy of the pho­tore­cep­tors and reti­nal pig­ment ep­ithe­li­um. While our re­sults demon­strate that the ge­net­ic risk vari­ants con­tribut­ing to AMD af­fect cone pho­tore­cep­tors, they are as­so­ci­at­ed with ad­di­tion­al cell types, high­light­ing the im­por­tance of glial and vas­cu­lar cells in dis­ease patho­gen­e­sis. […] our da­ta pre­dicts that these cell types pref­er­en­tial­ly con­tribute to the patho­gen­e­sis of AMD, and pro­vide ge­net­ic con­text to tar­get these cell types for the de­vel­op­ment of new dis­ease ther­a­pies.

By pin­point­ing cell types that are re­spon­si­ble for the de­te­ri­o­ra­tion, sci­en­tists can in­ves­ti­gate new tar­gets lead­ing to po­ten­tial­ly new types of ther­a­py, added Bri­an Hafler, co-se­nior au­thor and as­sis­tant pro­fes­sor of oph­thal­mol­o­gy and vi­su­al sci­ence and of pathol­o­gy at Yale.

Each sub­pop­u­la­tion of cells they iden­ti­fied ap­peared to be marked by high­er ex­pres­sion of sub­type-spe­cif­ic mark­ers, in­clud­ing FOS, FTL and COL4A3. Oth­er genes such as GAPDH and UBC were ubiq­ui­tous­ly ex­pressed, they not­ed.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

Leen Kawas, Athira CEO

Biotech founder placed on leave as $400M Alzheimer's start­up idea comes un­der scruti­ny

Athira Pharma, the Alzheimer’s biotech that emerged out of obscurity last year and raised nearly $400 million for a dark-horse approach to treating neurodegeneration, has found itself in sudden turmoil.

On Tuesday evening, the company released a terse statement announcing that CEO and founder Leen Kawas had been placed on administrative leave while an independent review board investigated “actions stemming” from her doctoral research at Washington State University. Mark Litton, who joined the company as COO two years ago, will take over day-to-day operations, they said.

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Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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FDA's con­tro­ver­sial Aduhelm de­ci­sion leaves ALS pa­tients feel­ing spurned

The FDA’s controversial approval of Biogen’s Aduhelm drug for Alzheimer’s disease has been met with fierce resistance from all corners of the biopharma industry, but few seem to be as upset with the decision as ALS patients and advocacy groups.

For all that’s already been written and discussed about the agency’s announcement, from the drug’s exorbitantly high price of $56,000 per year to criticism over lowered standards, ALS patients see something more. ALS patients and associations say they largely regarded Aduhelm’s approval as a bittersweet double standard: happy that those with Alzheimer’s have a new drug available, but questioning how the FDA evaluated Biogen’s drug compared to the experimental programs being studied for their own disease.

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Spring reg­u­la­to­ry agen­da: What’s com­ing soon-ish from the FDA

The FDA’s lack of a permanent commissioner does not seem to be halting its progress to propose and finalize dozens of new regulations, with the latest batch covering everything from adverse event reporting to supplemental application submissions to annual reports for INDs.

Overall, FDA expects to release more than 40 new proposed regulations and finalize another 24 in the coming months and years.

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Christian Hogg, Hutchmed CEO

Hutchmed files for $600M+ IPO in Hong Kong as lead on­col­o­gy drug su­r­u­fa­tinib awaits FDA's good graces

In oncology, a flush of Chinese-developed drugs has the biopharma industry rethinking the poles of power in R&D as the blossoming nation continues to make a name for itself and pick up bundles of cash in the process. Now, as its lead drug faces a pivotal FDA review, the company formerly known as Chi-Med is planting its flag on home soil with a massive public offering.

Hutchmed — recently renamed from Chi-Med, or Hutchison China MediTech — will look to raise $603 million as part of a Hong Kong IPO that serves as a homecoming of sorts for the Chinese-based oncology player, which has listed on Nasdaq since 2016.