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October 7, 2025

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The Fall Biotech Watchlist: 12 High-Stakes Decisions Ahead

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Contributing Writer

By Ryan Flinn

Overview

From one-time gene fixes to new cancer drugs, here are the breakthroughs and decisions to watch for in Q4.

The Fall Biotech Watchlist: 12 High-Stakes Decisions Ahead Copy

Every few months, the biotech industry faces a fresh round of judgment days—when pivotal trial data, FDA rulings, or long-awaited scientific updates can make or break a company’s future. As 2025 draws to a close, two new analyst reports from TD Cowen and Jefferies spotlight the milestones to watch in the final quarter of the year, from one-time gene edits to next-generation cancer therapies that could reshape treatment options.

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Biggest Scientific Breakthroughs

1. Intellia’s one-time gene edit for a swelling disorder

Intellia is developing a CRISPR treatment that aims to turn off a faulty gene causing dangerous swelling attacks in hereditary angioedema, which could offer lasting protection without repeated medicine. The company may share extended early-stage data before the end of the year, providing additional insights into how long the single-dose treatment continues working and whether it can eliminate the need for chronic therapy.

2. Cabaletta’s immune system reset for lupus

Researchers are adapting cancer-fighting cell therapy, called CAR-T, to reboot the immune system in autoimmune diseases like lupus and myositis, representing a major shift in how such conditions might be treated. The company said it will provide early-stage data in these two conditions before the end of the year, potentially validating whether reprogramming immune cells can provide long-term disease remission without ongoing medication.

3. Syndax’s precision approach to leukemia

The FDA will decide by October 25 whether to approve a first-in-class pill for a specific genetic form of acute myeloid leukemia, a blood cancer. Approval would mark the first therapy designed specifically for this mutation, potentially offering personalized treatment for patients who currently receive only standard chemotherapy.

Broadest Real-World Patient Impact

4. Celcuity’s breast cancer combination

A late-stage study readout in the fourth quarter could add another treatment option for the most common form of breast cancer, helping many women extend survival and potentially avoid more toxic therapies. The trial is testing whether adding Celcuity’s drug to standard hormone therapy improves outcomes in patients with different genetic profiles.

5. Exelixis’ colorectal cancer survival data

Results expected in Q4 may show longer overall survival for patients with colorectal cancer, one of the deadliest cancers worldwide. The late-stage trial is evaluating whether Exelixis’ drug combined with immunotherapy beats the current standard in second-line treatment, a result that could reshape treatment guidelines if successful.

6. Agios’ oral medicine for sickle cell disease

Topline results from a late-stage trial in Q4 will reveal whether an oral pill can reduce painful crises and hospital visits for sickle cell patients, giving them an easier alternative to transfusions or gene therapy. If successful, this approach could provide the first convenient daily treatment option for a disease that predominantly affects Black patients.

Biggest Potential Market Movers

7. Praxis Precision’s epilepsy data

Praxis expects to release topline late-stage data for its epilepsy drug before the end of the year. Success in this large patient population could establish the company’s treatment as a major new epilepsy option.

8. Alector’s dementia therapy for rare genetic form

A late-stage study reading out in mid-Q4 will show whether restoring a missing brain protein can slow cognitive decline in frontotemporal dementia caused by genetic mutations, a rare inherited form that strikes people in mid-life. Positive results would represent the first disease-modifying treatment for any form of this dementia and validate Alector’s approach of replacing deficient proteins in genetic neurological diseases.

9. Biohaven’s first treatment for movement disorder

An FDA decision expected in Q4 could deliver the first approved treatment for spinocerebellar ataxia, a progressive condition that slowly robs people of coordination and balance. Approval would validate a new mechanism for treating neurodegenerative disease.

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