Cell Therapies
After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting some reimbursement prices. Commercial success remains mixed and some products have been withdrawn from the market.
Despite initial promise, a novel autologous cell transplant therapy for severe heart failure has faced multiple challenges in gaining full approval in Japan, potentially providing learnings for other developers of regenerative medicines.
In this week's podcast edition of Five Must-Know Things: global CEO remuneration; another late-stage loss for Merch KgaA; deals shift to smaller M&A, partnering; Bayer’s Parkinson’s bets bear fruit; and India looks to innovate CAR-T models.
The German major is kicking off Phase II trials for a cell therapy and a gene therapy that could be gamechangers for the progressive neurodegenerative disorder that affects more than 10 million people worldwide.
SanBio’s lead cell therapy asset has been on a bumpy journey to its global-first approval and while a nod has now come in Japan in a high-need indication, a commercial launch is conditional on additional data to establish product equivalence and manufacturing consistency.
UK-based Ochre Bio has signed its first major deal with Boehringer Ingelheim. Scrip talked to its co-founder and CEO, Jack O’Meara, about its human tissue-based drug discovery platform, its resulting RNA platform for liver disease and how the fledgling drug company's early work mirrored that seen in diagnostics.
As drug pricing continues its ascent among hot button issues, the multi-million-dollar price tags for one-and-done advanced therapies, their justification and reimbursement all remain unaligned.
More than 75 industry executives and experts gazed into the crystal ball and shared with Scrip their predictions around transformative technologies. Perhaps unsurprisingly, AI was the dominant theme – but gene editing, cell therapies and new targeting modalities were also among the highlights.
2023 saw a number of gene therapy companies go to the wall, but those that can combine clinical significance and commercial success can build sustainable businesses.
Anito-cel showed strong efficacy in heavily pretreated patients despite characteristics predicting poorer performance, but none of the atypical neurotoxicities that have appeared with other BCMA CAR-Ts.
Autolus presented event-free survival rates and other updated results at ASH from its Phase Ib/II FELIX clinical trial of obe-cel, a CD19 CAR-T therapy recently submitted for US FDA approval.
Heart disease seems impervious to cell therapy approaches. Is biomarker stratification the answer?
Center for Medicare and Medicaid Innovation (CMMI) deputy director Ellen Lukens talked about the goals and challenges of a new voluntary program the agency will test with state Medicaid programs.
Companies aiming to be fast followers in sickle cell disease gene therapy behind Vertex and bluebird have thinned out in 2023, and Beam may struggle to convince investors its base editing approach can compete.
Launched with an ambitious R&D vision for cell therapies two years ago, Sana is now having to narrow its focus while it awaits the first of its Phase I readouts.
The German group has invested $250m into a facility at its campus in Berkeley, CA to produce cell therapies from its own portfolio and for other companies in the space who are looking to tackle the perennial issue of manufacturing.
Biopharma fears that the Joint Clinical Assessment, a first step to harmonizing health technology assessments in the EU, is not fit for purpose for cell and gene therapies and will increase rather than eliminate market access delays.
A few years ago, investors were calling on Vertex to buy in late-stage innovation, but its focus on audacious drug development goals looks set to pay off with a groundbreaking CRISPR-based sickle cell disease cell therapy approaching the market. Its R&D head David Altshuler talked to Scrip about its approach.
Japanese major’s profit figures for fiscal year just ended will be hit by multiple accounting decisions related to sales, pipeline and partnerships.
The confluence of data science and molecular biology fuels expectations among biopharma industry leaders for another year of breakthrough innovation in human therapeutics. More than 70 executives and experts reveal what they are most excited about on the technology front.