Re­searchers led by Broad In­sti­tute gene edit­ing star David Liu have come up with a new use for prime edit­ing that can tack­le a buck­et of dis­eases with the same root cause, which they say opens up a more straight­for­ward path to ad­dress dozens or even hun­dreds of ge­net­ic dis­eases.

The am­bi­tious idea could, in the­o­ry, of­fer sweep­ing ways to treat ge­net­ic dis­eases like cys­tic fi­bro­sis, Tay-Sachs dis­ease and many rare dis­eases that short­en lives. If those dis­eases have treat­ments at all, they cur­rent­ly re­ly on com­pli­cat­ed or cost­ly ther­a­pies unique to each ail­ment.

Agios Phar­ma­ceu­ti­cals’ drug Pyrukynd hit on­ly one of the two pri­ma­ry end­points in its piv­otal tri­al in sick­le cell dis­ease, the com­pa­ny said Wednes­day, a sig­nif­i­cant set­back for the com­pa­ny's plans to ex­pand use of the treat­ment.

The com­pa­ny said that it would still take the da­ta to reg­u­la­tors, in the hope of adding sick­le cell to the pill’s la­bel. Pyrukynd is al­ready ap­proved for the rare dis­ease pyru­vate ki­nase de­fi­cien­cy, but sick­le cell is much more preva­lent and ap­proval could in­crease the drug’s sales con­sid­er­ably.

Its stock AGIO crashed by 50% on Wednes­day in re­sponse to the news.

Jeff Be­zos' in­vest­ment firm pitched in to a $106 mil­lion fi­nanc­ing for Proflu­ent, an AI-fo­cused biotech start­up that has a near­ly $1 bil­lion val­u­a­tion.

Proflu­ent was found­ed in 2022, ad­vanc­ing re­search that CEO Ali Madani had start­ed at Sales­force. He was build­ing large lan­guage mod­els to un­der­stand and process pro­tein se­quences in­stead of words.

The Berke­ley, CA-based start­up has ad­vo­cat­ed for bring­ing the so-called scal­ing laws in­to bi­ol­o­gy — or the con­cept that a mod­el's per­for­mance will re­li­ably im­prove when giv­en more da­ta and com­pute pow­er.

Proflu­ent had last raised $35 mil­lion in March 2024. The newest fi­nanc­ing was co-led by Be­zos Ex­pe­di­tions and Al­time­ter Cap­i­tal, a tech-fo­cused in­vest­ment firm. Forbes first re­port­ed the raise, and that the start­up com­mand­ed a val­u­a­tion of near­ly $1 bil­lion.