Bill Gates has a $100M and a 5-point strat­e­gy to end 15 years of fail­ure in Alzheimer’s R&D

Bill Gates is go­ing af­ter Alzheimer’s.

The soft­ware bil­lion­aire is in­vest­ing $50 mil­lion of his own mon­ey — not from the Gates Foun­da­tion — in­to the in­dus­try/gov­ern­ment backed De­men­tia Dis­cov­ery Fund, which set out a cou­ple of years ago to back some trans­la­tion­al work on new ap­proach­es to the dis­ease. And he tells Reuters that he’s ear­marked $50 mil­lion more to back new com­pa­nies on his own that have the po­ten­tial to tack­le some fresh ap­proach­es to a dis­ease that has de­feat­ed vir­tu­al­ly every­thing thrown at it in the last 15 years.

Most of the big Phase II­Is have looked to elim­i­nate two pos­si­ble caus­es of Alzheimer’s — amy­loid be­ta and tau — in a crude at­tempt to bend the curve of the dis­ease. But all the piv­otal work by Eli Lil­ly, J&J, Mer­ck and oth­ers has failed bad­ly over the years.

Gates wants to go in some new di­rec­tions.

In an in-depth blog post dis­cussing his in­vest­ment in the fund, which is man­aged by SV Life Sci­ences, Gates out­lined his own 5-part strat­e­gy to tak­ing a fresh ap­proach. His thoughts:

  • We are woe­ful­ly ig­no­rant of how this dis­ease de­vel­ops and what may be dri­ving it. Why are blacks and Lati­nos more like­ly to de­vel­op Alzheimer’s? No one knows. So it’s time to go back and do the ba­sic re­search to study caus­es and bi­ol­o­gy.
  • Let’s de­vel­op a re­li­able way — per­haps a blood test — to di­ag­nose Alzheimer’s ear­li­er. The on­ly sure-fire way to do it now is through an au­top­sy, which has some ob­vi­ous lim­i­ta­tions in terms of mount­ing clin­i­cal stud­ies.
  • Wouldn’t it be great if tau and amy­loid be­ta worked as a tar­get? (Gates is noth­ing if not sup­port­ive.) Yes, but he wants to fund some new ideas.
  • “If we could de­vel­op a process to pre-qual­i­fy par­tic­i­pants and cre­ate ef­fi­cient reg­istries,” Gates writes, “we could start new tri­als more quick­ly.”
  • Com­pile all the da­ta out there in one plat­form and let re­searchers ex­plore it for new clues to de­vel­op­ing drugs and di­ag­nos­tics. Not sur­pris­ing­ly for the Mi­crosoft founder, that’s Gates’ sweet spot and where he might fo­cus much of his at­ten­tion.

“My back­ground at Mi­crosoft and my (Gates) Foun­da­tion back­ground say to me that a da­ta-dri­ven con­tri­bu­tion might be an area where I can help add some val­ue,” he told Reuters.

Gates is in it for the long haul. He ex­pects it could quite like­ly be a decade or more be­fore some­thing sub­stan­tial comes out of this all. And as a re­sult it makes more sense to in­vest his own mon­ey rather than the foun­da­tion’s. He notes:

I’m mak­ing this in­vest­ment on my own, not through the foun­da­tion. The first Alzheimer’s treat­ments might not come to fruition for an­oth­er decade or more, and they will be very ex­pen­sive at first. Once that day comes, our foun­da­tion might look at how we can ex­pand ac­cess in poor coun­tries.

Bill Gates has one of those leg­endary tech rep­u­ta­tions all biotechs love to be as­so­ci­at­ed with. And Gates-backed biotechs have that ex­tra lus­ter that opens doors, rais­es cash and gets the at­ten­tion of the ma­jor play­ers. The Alzheimer’s R&D groups he will back over the com­ing years have a lot more to gain than his mon­ey.

Im­age: Bill Gates. WEC

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).