CRISPR trail­blaz­ers Zhang, Liu and Joung join forces to launch Beam with $87M and cut­ting-edge gene-edit­ing tech

Three of the founders be­hind the high pro­file gene-edit­ing com­pa­ny Ed­i­tas are tak­ing their sci­en­tif­ic prowess to a new ven­ture, launch­ing a start­up this week that ex­pands on the pi­o­neer­ing CRISPR tech the en­tire space is built on.

The new com­pa­ny — called Beam Ther­a­peu­tics — is step­ping out Mon­day with $87 mil­lion in launch mon­ey. In­vestors are back­ing true trail­blaz­ers in gene edit­ing, as Beam’s sci­en­tif­ic founders in­clude David Liu, now-se­r­i­al en­tre­pre­neur Feng Zhang, and J Kei­th Joung. All three are sci­en­tif­ic founders of Ed­i­tas, the CRISPR com­pa­ny launched in 2013 that now gar­ners a $1.7 bil­lion mar­ket cap.

Beam is tak­ing a to­tal­ly new ap­proach to gene edit­ing, hop­ing to tweak base pairs with­out ac­tu­al­ly cut­ting the strand of DNA or RNA. If CRISPR can be com­pared to scis­sors, then Beam’s base edit­ing tech is more like a pen­cil, Liu tells me, eras­ing a “bad” base and writ­ing in a good one.

The im­pe­tus for Beam’s launch was Liu’s re­search at Har­vard, which is now li­censed to the Cam­bridge start­up. The li­cense cov­ers two base edit­ing plat­forms: a C base ed­i­tor and an A base ed­i­tor. The C base one fea­tures Cas9 linked to a cy­ti­dine deam­i­nase to make C-to-T or G-to-A ed­its. The A base ed­i­tor has Cas9 linked to “an evolved form of adeno­sine deam­i­nase,” which can make A-to-G or T-to-C ed­its.

Liu’s DNA base edit­ing tech is just part of Beam’s pack­age, though. Zhang, the guy who just months ago helped launched a dif­fer­ent gene edit­ing start­up (Ar­bor), has joined forces with Liu, con­tribut­ing RNA base edit­ing tech­nol­o­gy from his lab that fea­tures Cas13 linked to an adeno­sine deam­i­nase that can ed­it A-to-G in RNA tran­scripts.

On top of li­cens­es to Liu and Zhang’s tech, Beam al­so has patents from Ed­i­tas. Beam’s CEO John Evans tells me the deal gives the start­up ex­clu­sive rights to IP li­censed to Ed­i­tas by Har­vard, Broad, and Mass­a­chu­setts Gen­er­al Hos­pi­tal, along with cer­tain Ed­i­tas tech­nol­o­gy. As part of that deal, Beam has an ex­clu­sive sub­li­cense to base edit­ing patents out of Liu’s lab and patents by MGH for CRISPR tech­nol­o­gy de­vel­oped in Joung’s lab.

Beam’s ap­proach to gene edit­ing is quite com­pelling for a num­ber of rea­sons. First, slic­ing DNA is a per­ma­nent change, and that isn’t al­ways a good idea.

“There are some states of dis­ease that don’t last a life­time, but are tran­sient,” Liu said. “For those cas­es, you may not want to ad­dress the dis­ease by mak­ing a per­ma­nent change to the DNA of cells.”

“Eras­ing and writ­ing in” ed­its with­out clip­ping could be in­cred­i­bly use­ful in these cas­es.  Liu said edit­ing mu­ta­tions in the nu­cle­obas­es of DNA and RNA has the po­ten­tial to “re­verse a large frac­tion — per­haps the ma­jor­i­ty — of point mu­ta­tions as­so­ci­at­ed with dis­ease.”

But what are the odds that it will work? Liu said he’s hap­py with ear­ly in­di­ca­tions. Af­ter first pub­lish­ing work on these base ed­i­tors back in 2016, dozens of re­searchers have tried their hand at the tech­nol­o­gy and saw suc­cess in bac­te­ria, fun­gi, in­sects, mice, and even hu­man em­bryos.

Beam wants to stay qui­et on which dis­eases its first tar­gets will tack­le but did men­tion that the com­pa­ny is ac­tive­ly work­ing on 10-15 tar­gets and is “en­cour­aged by ear­ly re­sults.” This Se­ries A fund­ing, which was led by F-Prime Cap­i­tal Part­ners and ARCH, should take the com­pa­ny “mul­ti­ple years,” Evans said.

“The length of that run­way gives us con­fi­dence that we can push for­ward quick­ly with lead pro­grams, but al­so take time to go deep on the sci­ence and build a broad pipeline in par­al­lel,” Evans said.

Evans has joined Beam from ARCH, where he was a part­ner. Al­though VCs of­ten sit in as tem­po­rary CEOs while a start­up hunts for a more per­ma­nent can­di­date, Evans said he’s in for the long haul at Beam. While still af­fil­i­at­ed with ARCH, Evans is join­ing Beam full time.

“At Agios, I saw the pow­er of pre­ci­sion med­i­cine to di­rect­ly cor­rect mu­ta­tions in leukemia,” Evans said. “That was a pow­er­ful ex­pe­ri­ence for me, and I want to do that again. With base edit­ing, I see a re­al po­ten­tial to do it again and again.”

Beam has plans to set up an of­fice in the heart of Cam­bridge’s Cen­tral Square neigh­bor­hood. It em­ploys 15 peo­ple to­day, and plans on hir­ing “sev­er­al more” in the near fu­ture.


Top Im­age: Feng Zhang, David Liu and J Kei­th Joung. BEAM THER­A­PEU­TICS

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Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

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