CRISPR pi­o­neer Feng Zhang co-founds a 'lim­it­less' biotech up­start with big plans for speed­ing new drug de­vel­op­ment

One of the key sci­en­tif­ic play­ers in­volved in the emer­gence of the in­cred­i­bly buzzy gene edit­ing tech CRISPR/Cas9 is back­ing a biotech start­up called Ar­bor Biotech­nolo­gies in Cam­bridge, MA, which just un­veiled what it be­lieves is a new, more ver­sa­tile CRISPR tech.

In a pa­per pub­lished to­day in Mol­e­c­u­lar Cell, two for­mer mem­bers of Feng Zhang’s lab — David Scott and Win­ston Yan — out­lined their dis­cov­ery of an en­zyme called Cas13d, which they say is con­sid­er­ably small­er and bet­ter than the rest of the Cas13 fam­i­ly of en­zymes, giv­ing it greater po­ten­tial in RNA surgery. And their pa­per was pub­lished on the same day as a sep­a­rate study out of Salk which cen­tered on the ex­act same en­zyme.

The biotech came out of stealth mode to­day with a $15.6 mil­lion round, which we’ve been track­ing, and a plan to em­ploy the new CRISPR sys­tem with a plat­form drug de­vel­op­ment tech built around ar­ti­fi­cial in­tel­li­gence, genome se­quenc­ing, gene syn­the­sis and screen­ing ef­forts to go about the busi­ness of find­ing new mol­e­cules. Us­ing the new CRISPR sys­tem to char­ac­ter­ize pro­teins, the group plans to play a role in the on­go­ing in­te­gra­tion of com­pu­ta­tion­al sci­ence in drug dis­cov­ery, look­ing to short­en de­vel­op­ment time­lines and im­prove on some in­cred­i­bly bad fail­ure rates.

In a call with me late Thurs­day, Scott and David Cheng, founder of the search en­gine at Ar­bor, em­pha­sized that the new pa­per on Cas13d was done en­tire­ly in-house.

“All of this is whol­ly an ef­fort at Ar­bor,” says Scott. “Feng is a co-founder, he is in­volved, but he is not an au­thor of the study, that was whol­ly per­formed in-house” at the 2-year-old biotech.

From the sum­ma­ry of the new pa­per:

The small size, min­i­mal tar­get­ing con­straints, and mod­u­lar reg­u­la­tion of Cas13d ef­fec­tors fur­ther ex­pands the CRISPR toolk­it for RNA ma­nip­u­la­tion and de­tec­tion.

At the same time, re­searchers at the Salk In­sti­tute al­so un­veiled their sci­en­tif­ic work on Cas13d, us­ing it to tai­lor RNA rather than DNA, where CRISPR made its rep.

“CRISPR has rev­o­lu­tion­ized genome en­gi­neer­ing, and we want­ed to ex­pand the tool­box from DNA to RNA,” said Salk’s Patrick Hsu. And like Feng Zhang, he be­lieves that Cas13d en­zymes are per­fect­ly suit­ed for the job, with im­pli­ca­tions for dis­ease caused by tox­ic RNA or im­prop­er­ly spliced RNA, the mes­sen­gers that trans­late DNA in­to pro­teins. Dubbed Cas­Rx, they pack­aged it in a virus and used it to tar­get the tox­ic tau known to clus­ter in the brains of Alzheimer’s pa­tients. Aimed at neu­rons, it worked in cells grown from a pa­tients with neu­rode­gen­er­a­tive dis­or­der fron­totem­po­ral de­men­tia, re­bal­anc­ing tau lev­els.

David Scott

It will like­ly take years to prove if these young rev­o­lu­tion­ar­ies — and a host of col­leagues op­er­at­ing in AI — are right, but they al­ready have the rapt at­ten­tion of every drug de­vel­op­ment or­ga­ni­za­tion on the plan­et.

David Walt of Har­vard and the Wyss In­sti­tute and co-founder of Il­lu­mi­na and Quan­ter­ix is cred­it­ed as a co-founder with Zhang along with Scott and Yan, who both worked in Zhang’s MIT lab. And they are pumped about the sci­en­tif­ic po­ten­tial.

“Ar­bor’s rev­o­lu­tion­ary plat­form ac­cel­er­ates the rate of dis­cov­ery and char­ac­ter­i­za­tion of new bio­mol­e­cules by or­ders of mag­ni­tude,” said Scott in a state­ment.

Win­ston Yan

“We are now at the cusp of be­ing able to con­vert se­quence da­ta in­to a cat­a­log of pro­tein func­tions. The pos­si­bil­i­ties are lim­it­less,” not­ed Yan.

Zhang was the prin­ci­pal sci­en­tist be­hind the emer­gence of Ed­i­tas, one of the orig­i­nal gene edit­ing star­tups to hit fol­low­ing the emer­gence of CRISPR/Cas9, a tech that sev­er­al biotechs are us­ing to ed­it out dis­ease in genes.

Kei­th Cran­dell of ARCH Ven­ture Part­ners, An­nie Ha­zle­hurst of Fari­dan Ven­tures, and sev­er­al pri­vate in­vestors are cred­it­ed with the round.


Im­age: Feng Zhang. MIT

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.