Endpoints News
Ocugen heads to Phase 3 with gene therapy to prevent vision loss Read in browser
Endpoints News
Thank you for reading, dupa dupackia!
basic
UPGRADE
M Tue W Th F
24 March, 2026
Drug Discovery Day 2026
AI has gone from buzzword to pipeline strategy — but are the results a mixed bag? We're talking to the researchers in the thick of it about what's moving the needle, what's stalling out and what comes next. Join us for a free virtual program, then continue the conversation at an in-person-only fireside and happy hour in Boston. Choose your pass.
presented by FUJIFILM
Fu­ji­film ex­pands glob­al life sci­ences foot­print through large-scale bio­man­u­fac­tur­ing in­vest­ments
top stories
1. Q&A: Embryo editing entrepreneur Cathy Tie closes one startup and begins another in biology’s most taboo frontier
2. Ocugen heads to Phase 3 with gene therapy to prevent vision loss from macular degeneration
3. Biotech investors will meet with congressional panel, pushing US competitiveness changes
4.
news briefing
Karyopharm’s mixed myelofibrosis data; Rezolute to seek FDA approval despite trial failure
5. Gilead bets $1.68B upfront on Ouro Medicines’ autoimmune program, with Galapagos expected to pitch in
6. Merck partners with Flagship's 'somatic genomics' startup to search for clues to IBD
7. Shionogi will pay $100M to buy out Apnimed's part of their sleep disorder partnership
8. UK biotech co-founded by Nobel laureate secures $86M for antibody program
more stories
 
Drew Armstrong
.

Ryan Cross has an interview today with one of biotech's most controversial figures. Cathy Tie has plans to edit embryos to prevent disease, and is arguing that the risks are worth breaking what has for years been an ethical red line for much of the field.

.
Drew Armstrong
Executive Editor, Endpoints News
@ArmstrongDrew
Cathy Tie, Origin Genomics founder
1
by Ryan Cross

Cathy Tie has an au­dac­i­ty more typ­i­cal of a tech start­up founder than a biotech ex­ec­u­tive. She dropped out of col­lege to start a ge­net­ic screen­ing com­pa­ny and lat­er found­ed a telemed­i­cine start­up. The 29-year-old has been on two Forbes “30 un­der 30” lists (Ed­i­tor’s note: Not al­ways a good thing!) and calls her­self “Biotech Bar­bie,” yet un­til re­cent­ly, she hadn’t been on the radars of most peo­ple in biotech.

That changed last year when she an­nounced her ap­par­ent mar­riage and sub­se­quent breakup with He Jiankui, the in­fa­mous sci­en­tist who was jailed in Chi­na for us­ing CRISPR to cre­ate the world’s first gene-edit­ed ba­bies in 2018. Tie’s re­la­tion­ship with a sci­en­tif­ic pari­ah was quick­ly fol­lowed by the launch in Au­gust of a US com­pa­ny called the Man­hat­tan Project.

Click here to continue reading
2
by Lei Lei Wu

Ocu­gen said it will take its gene ther­a­py for a form of vi­sion loss in­to a Phase 3 tri­al, af­ter re­port­ing mixed re­sults on Tues­day from a small­er study.

The gene ther­a­py is for pa­tients with ge­o­graph­ic at­ro­phy, an ad­vanced form of the eye dis­ease dry age-re­lat­ed mac­u­lar de­gen­er­a­tion that can lead to grad­ual loss of vi­sion as eye cells die. It's pitch­ing the treat­ment as an al­ter­na­tive to reg­u­lar in­jec­tions. Two of those in­jec­tions, Apel­lis Phar­ma­ceu­ti­cals’ Syfovre and Astel­las’ Iz­er­vay, were ap­proved in the US in 2023 for the eye con­di­tion.

In the Phase 2 tri­al de­scribed Tues­day, Ocu­gen en­rolled 51 pa­tients: One-third re­ceived a low­er dose of the gene ther­a­py, OCU410, one-third re­ceived a high­er dose, and one-third re­ceived no treat­ment. It re­port­ed no se­ri­ous side ef­fects in the tri­al.

Click here to continue reading
Endpoints webinars
Mar 24
11:30 am ET
From structure to selectivity: Federated AI for collaborative binding affinity prediction
Rhino Federated Computing
3
by Max Bayer

A group of biotech in­vestors will meet with mem­bers of a con­gres­sion­al com­mis­sion that's been charged with keep­ing the US com­pet­i­tive in biotech and pro­tect­ing na­tion­al se­cu­ri­ty, amid the rise of Chi­na's own life sci­ences in­dus­try and shift­ing pric­ing dy­nam­ics around the globe.

Known as the Na­tion­al Se­cu­ri­ty Com­mis­sion on Emerg­ing Biotech­nol­o­gy (NSCEB), the group was es­tab­lished in 2021, and has been hold­ing ses­sions with biotech stake­hold­ers, as well as hold­ing lis­ten­ing events around the US. On Tues­day, it will meet with at least sev­en biotech in­vestors, in­clud­ing rep­re­sen­ta­tives from JP Mor­gan, Pfiz­er, and sev­er­al oth­er ven­ture cap­i­tal firms, End­points News has learned.

Ac­cord­ing to an agen­da and at­tendee list re­viewed by End­points, the group will dis­cuss the com­mis­sion’s pri­or­i­ties, the im­pacts of the Trump ad­min­is­tra­tion’s push to low­er drug prices, FDA un­cer­tain­ty and IP pro­tec­tions.

Click here to continue reading
News Briefing: Quick hits from the biopharma web
4