Vivek Ra­maswamy’s Roivant Sci­ences has nabbed its lat­est clin­i­cal-stage pro­gram for biotech start­up #14 — Aru­vant — and this time they’re go­ing up against a cou­ple of the lead­ers in the gene ther­a­py world.

Their new drug is now dubbed RVT-1801, and it has a fa­mil­iar pro­file, in­sert­ing a mod­i­fied fe­tal he­mo­glo­bin gene in­to au­tol­o­gous stem cells through a lentivi­ral ve­hi­cle in or­der to treat sick­le cell dis­ease and β-tha­lassemia.

Ac­cord­ing to the wheel­ing and deal­ing ex­ecs at Roivant, their pro­gram is “the on­ly known clin­i­cal-stage gene ther­a­py to de­liv­er the gene en­cod­ing fe­tal he­mo­glo­bin, which has been mod­i­fied to op­ti­mize oxy­gen car­ry­ing ca­pac­i­ty and an­ti-sick­ling prop­er­ties.”

The drug is com­ing from the lab of Punam Ma­lik, di­rec­tor of the Cincin­nati Com­pre­hen­sive Sick­le Cell Cen­ter at Cincin­nati Chil­dren’s, which re­ceived an undis­closed up­front pay­ment and a mile­stone sched­ule. Ma­lik is sched­uled to re­veal more about their work at the up­com­ing ASH con­fer­ence next Mon­day. 

And Ma­lik, who trained as a physi­cian in In­dia, was sold on Roivant’s strat­e­gy for de­vel­op­ing new treat­ments for pa­tients around the world.

Roivant is al­so es­tab­lish­ing a non-prof­it group to im­prove ac­cess to drugs for pa­tients in the de­vel­op­ing world.

“A crit­i­cal rea­son why we chose to work with Roivant on this pro­gram was their au­then­tic com­mit­ment to pa­tients glob­al­ly,” said the sci­en­tist.

But they won’t be op­er­at­ing alone. At least 3 oth­er biotechs are al­so spear­head­ing gene ther­a­pies for these two ail­ments.

Just weeks ago CRISPR Ther­a­peu­tics $CR­SP and its part­ners at Ver­tex got a green light to start Phase I/II clin­i­cal work on CTX001, al­so de­signed to spur the pro­duc­tion of fe­tal he­mo­glo­bin in pa­tients’ stem cells. Blue­bird bio $BLUE has al­so been work­ing on a gene ther­a­py for these con­di­tions, while Sanofi’s Biover­a­tiv $BI­IV and Sang­amo $SG­MO had their IND ap­proved for a ri­val gene ther­a­py a few months ago. Glob­al Blood Ther­a­peu­tics, mean­while, is look­ing for an OK on their new sick­le cell dis­ease drug vox­elo­tor while No­var­tis and oth­ers have their own new meds in the pipeline.

Ra­maswamy has raised more than $3 bil­lion to back his grow­ing sta­ble of com­pa­nies, which now em­ploy more than 750 sci­en­tists and ex­ecs and sup­port staff around the world. 

Im­age: Vivek Ra­maswamy at BI­IS18 in Shang­hai End­points News

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.