Go-ahead given to ge­net­i­cally mod­ify hu­mans for the first time

Biotech firm wins ap­proval for Euro­pean tri­als of ‘molec­u­lar scis­sors’ to fight dev­as­tat­ing blood dis­or­der

The Sunday Telegraph - - News - By Sarah Knap­ton SCI­ENCE ED­I­TOR

HU­MANS will be ge­net­i­cally engi­neered in Europe for the first time this year, us­ing a ground­break­ing gene­s­plic­ing ther­apy af­ter reg­u­la­tors ap­proved tri­als.

The biotech com­pany Crispr Ther­a­peu­tics is hop­ing to cure the dis­ease beta tha­las­saemia, a dev­as­tat­ing blood dis­or­der that re­duces the pro­duc­tion of haemoglobin, the pro­tein which car­ries oxy­gen to cells.

With­out suf­fi­cient oxy­gen, suf­fer­ers can be left with bone de­for­mi­ties, se­vere anaemia, slow growth, fa­tigue and short­ness of breath.

The dis­ease is caused by a ge­netic mu­ta­tion in the HBB gene, but sci­en­tists are con­fi­dent that they can al­ter the body’s code to fix the prob­lem and re­store healthy lev­els of haemoglobin.

It will be the first hu­man dis­ease to be treated us­ing the tech­nol­ogy in the West. China has al­ready be­gun sim­i­lar tri­als, but does not have the same strict reg­u­la­tion as Europe or the US. The com­pany has yet to de­cide where the first trial will take place.

Bri­tish ex­perts hailed the new treat­ments as “very promis­ing”.

Prof Robin Lovell-Badge, Group Leader at Lon­don’s Fran­cis Crick In­sti­tute, said: “We will look back and think that this is the real be­gin­ning of gene ther­apy.”

Genome ther­apy has been around in a ba­sic form for around 30 years, with sci­en­tists ad­min­is­ter­ing miss­ing DNA to dam­aged cells so they are able to work more ef­fec­tively. Doc­tors have also used ge­net­i­cally engi­neered im­mune cells to keep pa­tients alive un­til a donor is found.

But Crispr, which stands for Clus­tered, Reg­u­larly In­ter­spaced, Short Palin­dromic Re­peat, could be a cheap and per­ma­nent so­lu­tion. It is based on a nat­u­ral de­fence mech­a­nism used by bac­te­ria, which carry in their DNA strands of ge­netic code be­long­ing to deadly viruses so that they can recog­nise them. When they spot a virus they re­lease an en­zyme which at­tacks it, snip­ping away this area of code.

Sci­en­tists have har­nessed this mech­a­nism to cre­ate “molec­u­lar scis­sors” which re­move mu­tated ar­eas of DNA.

Dur­ing the new trial the gene edit­ing will take place out­side of the body, with stem cells har­vested from pa­tients and then engi­neered in the lab.

Although some ther­a­pies in­volve in­sert­ing the cor­rect gene, the com­pany has taken a dif­fer­ent op­tion, seek­ing to boost foetal haemoglobin – the high lev­els of the pro­tein which ba­bies have – by re­mov­ing a gene that re­presses its pro­duc­tion in adults.

The engi­neered cells are then trans­fused back into the pa­tient’s bone mar­row, where they start pro­duc­ing high lev­els of haemoglobin, al­low­ing oxy­gen to once again flood cells.

Dar­ren Grif­fith, pro­fes­sor of ge­net­ics at the Univer­sity of Kent, said: “Ev­ery­thing I have seen sug­gests it’s very safe and ef­fec­tive. I think the [trial] re­sults will be pos­i­tive. And then we will be able to say, this is where it all be­gan.”

Sev­eral other com­pa­nies are hop­ing to de­velop sim­i­lar ther­a­pies, while the Univer­sity of Penn­syl­va­nia has be­gun re­cruit­ing for a new trial which seeks to use Crispr to treat peo­ple suf­fer­ing from var­i­ous forms of can­cer.

Chi­nese uni­ver­si­ties be­gan us­ing Crispr for can­cer in 2016. Dr Si­mon Wadding­ton, reader in Gene Trans­fer Tech­nol­ogy at Univer­sity College Lon­don, said: “I get the feel­ing that pub­lic sen­ti­ment has changed, in that ge­netic ther­a­pies are now more ac­cept­able.”

Prof Robin Lovel­lBadge said the tri­als would mark the be­gin­ning of gene ther­apy in Europe

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