A decade of proof-of-concept trials shows remyelination is achievable. The harder question, per a Cambridge neurologist's own assessment, is whether that biology will ever translate into measurable disability benefit.
Gene therapy treats disease at its source — the DNA …
Platform biotechs sell a repeatable method, not a single drug. …
A new documentary reopens the case, and the underlying lesson …
Biotech financing runs on staged proof: seed capital buys discovery …
Orphan drug designation is a formal FDA status for drugs …
The FDA has issued a complete response letter for Alvotech's …
Vertex Pharmaceuticals has presented the first clinical data for Casgevy in children ages 5-11 at the American Society of Hematology annual meeting, on December 6, 2025, in Orlando. All evaluated children in the sickle cell cohort remained free of vaso-occlusive crises for up to two years after…
Epigenetics is the field of study focused on changes in DNA that do not involve alterations to the underlying sequence, per NHGRI's Talking Glossary, which also calls the field epigenomics. The commercial and scientific interest sits in the platforms that read those changes at scale: sequencing-…
In vitro diagnostics are regulated as medical devices under different instruments in the US and EU: FDA classification and premarket review in America, and the dedicated European Regulation 2017/746 in Europe. The US boundary moved in 2025, when a federal court vacated the FDA's…
Medical device trials run on an Investigational Device Exemption, the instrument that "allows the investigational device to be used in a clinical study in order to collect safety and effectiveness data," per FDA — and all such evaluations, unless exempt, "must have an approved IDE before the…
A drug candidate reaches the clinic through an Investigational New Drug application, the exemption that lets a sponsor ship an unapproved drug to investigators. Per FDA, an IND is needed once a sponsor that has screened a molecule for "pharmacological activity and acute toxicity potential in…
A bioinformatics pipeline is a chained, software-defined series of steps that converts raw sequencing output into analyzed results, recording every tool version and parameter used. It exists to solve reproducibility: as the Nextflow authors wrote in Nature Biotechnology, the main source of…
Platform science in early-stage biotech is engineered, reusable biology — a delivery vector, an engineered binding domain, a data architecture — that one company claims can yield many programs rather than one. The claim is cheap; the validation is not. It arrives as published methods, reference…
A generic copies a small-molecule drug exactly. A biosimilar only comes close, because biology will not allow a perfect copy.
The FDA has approved Shionogi's Xocova (ensitrelvir) as the first oral post-exposure prophylaxis for COVID-19 in adults and adolescents 12 years of age and older, the company announced on June 1, 2026. The approval, covering use after contact with an individual who has COVID-19, came ahead of…
Eli Lilly said in May 2026 that it will invest an additional $4.5 billion at two of its three Lebanon, Indiana sites, lifting Indiana capital commitments above $21 billion over six years, per the company's announcement. A December 2025 decision to spend more than $6 billion on an Alabama API…
Molecular testing is moving to the point of care. Roche said on December 2, 2025 that its cobas liat Bordetella test received FDA 510(k) clearance and a CLIA waiver, with results in about 15 minutes, per the company. On August 6, 2026, Co-Diagnostics announced a dual 510(k) with a concurrent…
NIH's All of Us Research Program became the world's largest integrated genomic and electronic health record database with its June 30, 2026 release, which per the agency's announcement makes data from more than 747,000 participants available to scientists and includes more than 535,000 whole…
Lab automation in biotech has moved from liquid-handling convenience to process control: a nonlinear model predictive control system developed by Sartorius researchers increased continuous CHO perfusion production by 68% while holding viable cell density at or above 95%, per a September 23, 2026…