Biotech­nol­o­gy is en­ter­ing a dy­nam­ic new phase, de­fined by trans­for­ma­tive sci­ence and strong cap­i­tal in­vest­ment. De­spite on­go­ing in­vestor con­cerns and broad­er chal­lenges in the health­care sec­tor, such as pol­i­cy un­cer­tain­ty and tar­iffs, cer­tain cut­ting-edge ad­vance­ments are de­fy­ing the trend. RNA edit­ing and ra­di­oli­gand ther­a­py are two of the ther­a­peu­tic ar­eas gen­er­at­ing re­newed ex­cite­ment and op­por­tu­ni­ties for in­vest­ment, as biotech com­pa­nies seek to find new treat­ments for ge­net­ic dis­or­ders and can­cer.

RNA edit­ing is rewrit­ing the rules of med­i­cine and is emerg­ing as a high-po­ten­tial fron­tier in biotech. A more tem­po­rary coun­ter­part to DNA gene edit­ing, RNA tweaks mol­e­c­u­lar mes­sages with­out touch­ing DNA. “Think of DNA as the hard­ware and RNA as the op­er­at­ing soft­ware,” ex­plains Kris Elverum, CEO of AIR­NA, a biotech com­pa­ny.

This re­versible ap­proach has po­ten­tial for treat­ing ge­net­ic dis­or­ders, neu­rode­gen­er­a­tive dis­eases, pain and car­diometa­bol­ic con­di­tions. AIR­NA is ad­vanc­ing a new treat­ment for al­pha-1 an­tit­rypsin de­fi­cien­cy (AATD), a rare lung and liv­er dis­or­der, by ad­dress­ing the un­der­ly­ing gene mu­ta­tion. Elverum de­scribes AIR­NA’s RNA edit­ing as “the tech­nol­o­gy that can unique­ly tack­le this con­di­tion, which has few vi­able treat­ment op­tions avail­able to­day”. As a less per­ma­nent al­ter­na­tive to DNA edit­ing, it can al­so be used to pro­vide pos­i­tive ef­fects on health. As Elverum ex­plains: “We’re pi­o­neer­ing a new field of RNA edit­ing. By study­ing hu­man and ge­net­ic evo­lu­tion, we can iden­ti­fy ben­e­fi­cial ge­net­ic vari­ants and safe­ly in­tro­duce them in­to pa­tients. For com­plex dis­eases, the goal isn’t al­ways to elim­i­nate some­thing from the body. In­stead, RNA edit­ing al­lows us to fine-tune the body’s ‘soft­ware’ to cre­ate pos­i­tive out­comes.”

The suc­cess of ear­ly clin­i­cal tri­als of RNA edit­ing has sparked an in­vest­ment race, with da­ta from Wave Life Sci­ences’ ear­ly hu­man tri­al show­ing proof of mech­a­nism (proof that a drug can­di­date reach­es its tar­get or­gan) with a sin­gle dose. Noël Brown, Man­ag­ing Di­rec­tor and Head of Biotech­nol­o­gy In­vest­ment Bank­ing at UBS, be­lieves ex­cite­ment over the trans­la­tion of the pre­clin­i­cal da­ta to hu­man re­sults – which is of­ten not the case for many drugs – has dri­ven this up­surge in in­ter­est. He al­so points out that in­vestors have a “high­er lev­el of com­fort” with the tech­nol­o­gy, ow­ing to its re­versibil­i­ty com­pared with DNA edit­ing.

An­oth­er ground­break­ing treat­ment gen­er­at­ing ex­cite­ment among in­vestors is ra­di­oli­gand ther­a­py, which rep­re­sents a new, tar­get­ed weapon in the fight against can­cer. While tra­di­tion­al ra­dio­ther­a­py de­liv­ers treat­ment to the tu­mour while al­so af­fect­ing the sur­round­ing healthy tis­sue, ra­di­oli­gand ther­a­py us­es “tar­get­ed mis­siles” to de­liv­er ra­di­a­tion straight to in­di­vid­ual tu­mour cells, spar­ing healthy tis­sue and less­en­ing the side ef­fects. Al­ready im­prov­ing out­comes for prostate and neu­roen­docrine can­cers, this next-gen­er­a­tion ap­proach is now be­ing test­ed in a wider range of ad­vanced and metasta­t­ic tu­mours with lim­it­ed op­tions, lead­ing a new wave in pre­ci­sion on­col­o­gy treat­ment.

Brown high­lights that ma­jor play­ers – in­clud­ing No­var­tis, Bay­er, and Eli Lil­ly – are strate­gi­cal­ly ex­pand­ing their port­fo­lios by ac­quir­ing ra­di­oli­gand ther­a­py com­pa­nies with iso­tope man­u­fac­tur­ing ca­pa­bil­i­ties and cre­at­ing com­pet­i­tive bar­ri­ers in this niche in­dus­try.  He notes that a ma­jor hur­dle could arise, how­ev­er, as chal­lenges in the avail­abil­i­ty of iso­topes may ini­tial­ly re­strict the com­mer­cial roll­out of ra­di­oli­gand ther­a­pies. How­ev­er, he is san­guine. “As has his­tor­i­cal­ly been the case, de­mand such as this will ul­ti­mate­ly be met by new sup­pli­ers lever­ag­ing new tech­nolo­gies, so ex­pect to see new break­throughs in iso­tope man­u­fac­tur­ing as well.”

Al­though 2024 saw a spike in ven­ture cap­i­tal fund­ing in the sec­tor, there was a slow­down in the first half of 2025 caused by on­go­ing con­cerns over pol­i­cy and reg­u­la­tion.

How­ev­er, RNA edit­ing and ra­di­oli­gand ther­a­py are pro­vid­ing a glim­mer of hope that the biotech sec­tor is re­bound­ing, with re­cent large, high-qual­i­ty IPOs and ma­jor deals such as As­traZeneca’s an­nounce­ment of its $2bn ac­qui­si­tion of Fu­sion Phar­ma­ceu­ti­cals in March 2024 and Bris­tol My­ers Squibb’s $4.1bn pur­chase of Rayze­Bio the month be­fore. In RNA edit­ing, there have been few­er large deals, but part­ner­ships such as those be­tween Wave and GSK, and Pro­QR Ther­a­peu­tics’ bil­lion-dol­lar col­lab­o­ra­tion with Eli Lil­ly in­di­cate that these ther­a­pies are set to fol­low a sim­i­lar eco­nom­ic tra­jec­to­ry to that of CRISPR gene edit­ing.

Such deals show that the mar­ket is ready to di­gest new eq­ui­ty and is open for busi­ness, Brown says, un­der­scored by grow­ing in­vestor ex­cite­ment for break­through ther­a­pies such as RNA edit­ing and ra­di­oli­gand ther­a­py. In­vest­ments are clear­ly fol­low­ing ad­vance­ments that promise to dis­rupt the mar­ket and change the ther­a­peu­tic par­a­digm for a range of con­di­tions, po­si­tion­ing the biotech sec­tor for sus­tained growth dri­ven by in­no­va­tion.

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