NIH re­searchers re­port an­i­mal mod­el val­i­da­tion for stem cell-based ap­proach to ad­vanced dry AMD

A group of NIH re­searchers say they have laid the ground­work need­ed to tack­le the root cause of an ad­vanced form of age-re­lat­ed mac­u­lar de­gen­er­a­tion by re­pro­gram­ming adult blood cells in­to reti­nal cells in rat and pig mod­els. And now they plan to move that in­to an his­toric break­through ef­fort in hu­mans.

The sci­en­tists from the NIH’s Na­tion­al Eye In­sti­tute set out to pre­vent blind­ness in ge­o­graph­ic at­ro­phy, or ad­vanced dry AMD. Reti­nal pig­ment ep­ithe­lial cells nur­ture pho­tore­cep­tors; when they die (usu­al­ly in the ear­ly stage of ge­o­graph­ic at­ro­phy) the pho­tore­cep­tors lose pro­tec­tion and even­tu­al­ly die as well, ul­ti­mate­ly re­sult­ing in blind­ness. With the re­sults and pro­to­cols de­tailed in a Sci­ence Trans­la­tion­al Med­i­cine pa­per to­day, they are hop­ing to set the stage for a first-in-hu­man study.

The cells used in the au­tol­o­gous treat­ment goes through three stages: They be­gin as a pa­tient’s blood cells, get con­vert­ed in­to in­duced pluripo­tent stem cells (iP­SCs), and are fi­nal­ly pro­grammed to be­come reti­nal pig­ment ep­ithe­lial (RPE) cells and thin­ly lay­ered on a scaf­fold. RPE cells nur­ture pho­tore­cep­tors; their death marks the ear­ly stage of ge­o­graph­ic at­ro­phy, al­so known as ad­vanced dry AMD.

By patch­ing in iP­SC-de­rived RPE cells to re­place the dy­ing ones, re­searchers the­o­rized they could keep the pho­tore­cep­tors — light-sens­ing cells — func­tion­ing prop­er­ly, thus main­tain­ing vi­sion.

As far as they could tell, it worked.

Kapil Bhar­ti, lead in­ves­ti­ga­tor for the an­i­mal study

“Ten weeks af­ter the hu­man iP­SC-de­rived RPE patch­es were im­plant­ed in the an­i­mals’ reti­nas, imag­ing stud­ies con­firmed that the lab-made cells had in­te­grat­ed with­in the an­i­mal reti­na,” a re­lease reads. The lab-made cells al­so grew ma­ture enough to keep the pho­tore­cep­tors alive and healthy.

The re­searchers al­so checked to make sure the RPE cells they grew had no ge­net­ic mu­ta­tions linked to can­cer growth, a “key con­cern with any stem cell ther­a­py.”

The lead­ing cause of blind­ness among the el­der­ly af­fect­ing more than 5 mil­lion world­wide, ge­o­graph­ic at­ro­phy has once in­spired megablock­buster dreams at Roche for its eye drug lam­pal­izum­ab — which was qui­et­ly scraped af­ter fail­ing both Phase II­Is. Ken­tucky-based biotech Apel­lis has more en­cour­ag­ing da­ta with its com­ple­ment C3 in­hibitor. With an IND pend­ing, stem cell-based ther­a­py will soon be­come an­oth­er can­di­date to claim the first ap­proved treat­ment for the dis­ease.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Caught in a stand­off with its con­tract man­u­fac­tur­er over Covid-19 vac­cine, In­ovio files suit in an at­tempt to break free while ri­vals race ahead

Inovio was one of the first vaccine developers to snag attention for a jab that their execs said promised to end the Covid-19 pandemic. Using their own unique DNA tech, CEO Joseph Kim said it took just 3 hours to work it out.

But while rivals are racing to the finish line with ambitious plans to make vast quantities of their vaccines with billions of dollars of deals, Inovio is still stuck at the starting line on manufacturing.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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