Daily biotech news, content, and exclusive interviews.
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: Robert MacLaren is a professor of ophthalmology at Oxford, and Co-Founder of @BeaconTx_, which just scored a phase 3 win on its gene therapy for X-linked retinitis pigmentosa.
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: @GenomicsEngland Chair Nicola Blackwood describes why having a robust genomics effort is an important pairing to developing new therapies for rare diseases. @OHRareDisease
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: Sarah Tabrizi of University College London discusses her experience as an investigator on UniQure's Huntington's Disease gene therapy, AMT-130. @OHRareDisease
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: @ByAmyBrown asked @UrnovFyodor what his dream for genetic medicines would be for the year 2030.
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: "If you have a pizza place and you change the topping, you don't re-certify the oven."
@UrnovFyodor at @OHRareDisease's Symposium 2026 on how to lower the cost of gene therapies for patients.
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: At @OHRareDisease's Symposium 2026, Sir John Bell discusses the rare disease space and steps he thinks are needed to make the UK more competitive.
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: At @OHRareDisease's Symposium 2026, Co-Director Matthew Anderson discusses how to help get rare disease medicines funded and paid for.
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𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞: Today BiotechTV is at the @OHRareDisease's Symposium 2026 in Oxford, UK. Director Matthew Wood tells us about the event.
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𝐂𝐨𝐦𝐩𝐚𝐧𝐲 𝐍𝐞𝐰𝐬: @BeaconTx_'s gene therapy for X linked retinitis pigmentosa succeeded on its primary endpoint of helping patients to see better on low luminance visual acuity in a phase three study.
Full video: biotechtv.com/post/beacon-th…
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𝐁𝐢𝐨𝐕𝐞𝐧𝐭𝐮𝐫𝐞 𝐕𝐨𝐢𝐜𝐞𝐬: @blackstone's Kiran Reddy on neuroscience's renaissance and Blackstone's commitment to life sciences.
Watch the full discussion: biotechtv.com/post/kiran-red…
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𝐇𝐂𝐖 𝟐𝟎𝟐𝟔: As it approaches a BLA filing for its viral immunotherapy in prostate cancer, @CandelTx has published its pivotal phase 3 data in Lancet Oncology and presented extended data at AUA. $CADL
Full video: biotechtv.com/post/candel-th…
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𝐇𝐂𝐖 𝟐𝟎𝟐𝟔: InflaRx designed izicopan from the start with the goal of having a differentiated, best in class profile vs the first marketed C5aR inhibitor. Now more than ever this could be opportune. $IFRX
Full video: biotechtv.com/post/inflarx-h…
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𝐇𝐂𝐖 𝟐𝟎𝟐𝟔: @genfit_pharma has developed a blood based test for MASH - its CEO believes the MASH market has arrived. "If you believe in Mash, you have to believe in a diagnostic."
Full video: biotechtv.com/post/genfit-hc…
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𝐇𝐂𝐖 𝟐𝟎𝟐𝟔: @scribetx which represents the next generation of CRISPR, had its IPO in July - the first clinical program targets PCSK9. With Co-Founder & CEO @BenjaminLOakes. $SCTX
Full video: biotechtv.com/post/scribe-th…
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𝐇𝐂𝐖 𝟐𝟎𝟐𝟔: With a $6B valuation, three approved products, up to $700M in revenue guidance this year, and a sizable acquisition under its belt, @mirumpharma has become a rare disease leader. $MIRM
Full video: biotechtv.com/post/mirum-pha…
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𝐇𝐂𝐖 𝟐𝟎𝟐𝟔: With an in-human proof of concept in hand from a rare condition, @PrimeMedicine is ready to go broader in new indications. $PRME
Full video: biotechtv.com/post/prime-med…
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