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Venture capitalist Ron Conway says he has 'rare' cancer
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The opportunities and challenges facing rare disease therapies ...
To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024. The panel comprised: Jean-Philippe Combal, CEO of Vivet Therapeutics; Dr Jörg Thomas Dierks, CEO of Neuraxpharm; Catherine Pickering, CEO of iOnctura; Jeremy Skillington, CEO, of Poolbeg Pharma; and Miquel Vila-Perelló, CEO and co-founder at SpliceBio. What does the landscape for rare disease therapies look like in 2024? Jean-Philippe Combal (JPC): There are global challenges with financing of rare and ultra-rare disease therapies, while in the EU there are uncertainties as to whether payers are able to assess the value of uncertain long-time outcomes. It’s going to be increasingly important to create patient support frameworks to win over payers and collaborate with regulators to find early clinical benefit biomarker approaches for accelerated approvals. Miquel Vila-Perelló (MVP): We are off to an excellent start, with the approval of three orphan drugs in three different rare disease areas. Hopefully, we can sustain the trend observed in the last decade, whereby approximately 50% of approved drugs are orphan and aimed at rare diseases. Jeremy Skillington (JS): In 2024, advancements in rare disease therapies continue, with increased focus on precision medicine, gene therapies, and personalised treatment approaches. Research collaborations and innovative technologies play crucial roles in addressing the unique challenges of rare diseases. Access to these therapies may improve, but affordability and availability remain ongoing concerns. How significant was the first FDA approval of a CRISPR gene therapy in late 2023, and what other promising and cutting-edge developments are on the horizon? JPC: It was a turning point, but there is much more to be done. The ex-vivo CRISPR-based therapy adoption by patients will be an important next step, while the next cutting-edge step will be in-vivo CRISPR-based therapy - likely for a more limited number of indications. MVP: It was fantastic news for patients and an endorsement to all gene therapy modalities. The approval demonstrates the pace at which gene therapies are being developed and the clear unmet medical need (10 years from company foundation to first approval). Another development is the recent reports of AAV gene therapies capable of restoring hearing in patients born with mutations in the gene otoferlin. What’s significant is ...
Venture capitalist Ron Conway says he has 'rare' cancer
Ron Conway, a longtime venture capitalist and major donor to Democratic causes, said Friday that he has a "rare" form of cancer and is starting treatment for it immediately. Conway, founder of SV Angel, a San Francisco-based venture capital firm, said he will step back from his company, but still remain involved at times. "SV Angel has a deep, experienced team that remains fully focused ...
Tech investor Ron Conway says he has a rare form of cancer
Ron Conway, the influential Silicon Valley investor and longtime San Francisco political force, said Friday that he has been diagnosed with a rare form of cancer and will begin treatment immediately.
