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Pre­sent­ing Your Gene Ther­a­py Man­u­fac­tur­ing Plan to In­vestors: What They Ex­pect in 2026 and How to De­liv­er It
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1. A ‘cure’ for five blood disease patients suggests Chinese genetic medicine can compete globally
2. Biotech VCs ramp up checks on new bets after years of focusing on existing portfolios
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Vivatides raises Series A for R&D; Wegovy drops cold chain in EU; Gilead picks up $45M Kymera option
4. China-based CAR-T company Oricell raises $40M more as it looks to go public
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Drew Armstrong
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A Chinese biotech has — at least so far — cured five patients with the blood disease beta thalassemia using a gene editing tool it developed itself. A potentially cheaper, safer, more effective gene therapy from China poses a threat to American dominance in the area.

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Drew Armstrong
Executive Editor, Endpoints News
@ArmstrongDrew
1
by Ryan Cross

Five peo­ple with be­ta tha­lassemia who had their blood stem cells ge­net­i­cal­ly al­tered no longer re­quire reg­u­lar blood trans­fu­sions to stay healthy. Their Chi­nese-made treat­ment is like­ly to be cheap­er, and per­haps safer and more ef­fec­tive, than its Amer­i­can coun­ter­parts.

De­vel­oped by Cor­rect­Se­quence Ther­a­peu­tics, the ther­a­py em­ploys a nov­el form of CRISPR base edit­ing de­signed to change a sin­gle let­ter of DNA with al­most no un­want­ed and po­ten­tial­ly dan­ger­ous off-tar­get ed­its. The Shang­hai-based com­pa­ny told End­points News that the first pa­tient treat­ed has now been trans­fu­sion-free for near­ly two and a half years.

The study of the five pa­tients, pub­lished Wednes­day in Na­ture, ar­rives as in­ter­est grows among West­ern drug­mak­ers in ex­per­i­men­tal ther­a­pies made in Chi­na. And the new study sug­gests Chi­na will con­tin­ue to be­come a big­ger com­peti­tor even for the most cut­ting-edge med­i­cines.

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TOP R&D TRENDS IN 2026:
John Carroll’s R&D 15 report is back — and this year, he’s bringing some of the industry’s top research chiefs into the conversation to break down what the data means, what’s shifting, and where biopharma R&D is headed. Join us to find out what comes next.
2
by Kyle LaHucik

Biotech ven­ture cap­i­tal­ists are em­brac­ing risk again, sig­nal­ing a re­ver­sal from the past few years.

VCs are plac­ing more bets on flashy new biotech op­por­tu­ni­ties in the first quar­ter of 2026. About 65% of in­vest­ments by a group of the most ac­tive pri­vate biotech in­vestors went in­to new port­fo­lio com­pa­nies, ac­cord­ing to a quar­ter­ly re­port from bankers at William Blair.

It’s a stark con­trast from the pre­vi­ous sev­er­al years of belt-tight­en­ing, in which in­vestors’ dol­lars were fo­cused on ex­ist­ing port­fo­lio bets — a low­er-risk strat­e­gy.

“The qual­i­ty of deals we’re see­ing now, the lev­el of in­no­va­tion, it’s as high as I’ve seen it in the last cou­ple years,” Sam­sara Bio­Cap­i­tal’s Mike Dybbs said in an in­ter­view. “And the deals that we’re see­ing are rea­son­ably and very at­trac­tive­ly priced. It’s that com­bi­na­tion of ‘OK, port­fo­lio is in good shape’ and this flood of re­al­ly in­ter­est­ing, ex­cit­ing com­pa­nies.”

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News Briefing: Quick hits from the biopharma web
3
by ENDPOINTS

New Chi­na-US biotech gets $54M: Vi­vatides Ther­a­peu­tics raised the Se­ries A for its pre­clin­i­cal-stage R&D in siR­NA and an­ti­sense oligonu­cleotides aimed at reach­ing tis­sues be­yond the liv­er. Vi­vatides’ in­vestors in­clude Qim­ing Ven­ture Part­ners, High­light Cap­i­tal, Apri­cot Cap­i­tal and oth­ers. Qim­ing has backed mul­ti­ple promi­nent Chi­nese and US biotechs, such as Struc­ture Ther­a­peu­tics, In­sil­i­co Med­i­cine and Can­did Ther­a­peu­tics. — Kyle LaHu­cik

No­vo Nordisk’s obe­si­ty shot no longer needs cold chain de­liv­ery in the EU: A la­bel up­date means the in­ject­ed form of We­govy is now the first GLP-1 weight loss ther­a­py that can be kept at tem­per­a­tures up to 86 de­grees Fahren­heit for up to 48 hours. This could sim­pli­fy the drug’s dis­tri­b­u­tion and cut de­liv­ery costs. — Eliz­a­beth Cairns

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