'Futuristic' treatments come to fruition with cell and gene therapies

One-time treatments that could functionally cure rare diseases. Cyborg T cells trained to kill cancer. A biological hacking method that could allow scientists to rewire errant genes from within. This year, a host of moonshot technologies matured, stoking hopes for real-world applications.

The world of gene therapy--in which single-dose treatments correct debilitating defects--enjoyed something of a renaissance in 2014. Strong clinical results from leaders in the once-maligned field spurred renewed optimism, helping a new generation of startups secure millions in venture financing to develop their next-generation approaches to the field.

And that led to something of a trickle-up phenomenon in the industry, as the innovations of biotechs and academics convinced the world's biggest players to give the field a second look. Now Bayer, Pfizer ($PFE), Biogen Idec ($BIIB) and Astellas are among the many companies toiling in gene therapy, joining high-profile biotechs like bluebird bio ($BLUE) and uniQure ($QURE).

Cell therapy has had a somewhat similar history, with issues of safety, deliverability and manufacturing troubling the development of treatments that infuse living cells to treat disease. This year, however, a new approach called CAR-T made headlines with stellar clinical results in cancer. CAR-T therapies are made through a process in which scientists remove T cells from a patient's blood and equip them with targeting mechanisms called chimeric antigen receptors (CARs), which seek out and bind to proteins expressed by cancer cells. The resulting cells are reinjected into the patient, at which point they track down malignancies and attack them as they would any commonplace infection.

Such treatments have the potential to change the standard of care in certain blood cancers, and CAR-T developers--led by Novartis ($NVS) and Juno Therapeutics ($JUNO)--believe their therapies have wide potential in solid tumors, too. The clinical and commercial promise of CAR-T has lured a cadre of drugmakers into the field, including Celgene ($CELG), Pfizer, Johnson & Johnson ($JNJ), Kite Pharma ($KITE) and Bellicum Pharmaceuticals ($BLCM).

And then there's gene editing, an early-stage new technology that researchers say has staggering potential. The technology centers on short, repetitive bits of genetic code and a homing protein that can shuttle them around the body. The sequences, called CRISPRs, can be imposed on any location in the genome by hitching them to the Cas9 protein, which binds to specific RNA molecules. By crafting and deploying custom CRISPRs, scientists believe they can develop a naturalistic method of correcting defective genes, getting at the underlying causes of a broad range of diseases.

For evidence of CRISPR/Cas9's promise, look no further than its attendant battle over intellectual property. Many of the field's pioneers joined together to form Editas Medicine, a 2014 Fierce 15 honoree, but a breakup of co-founders led Berkeley's Jennifer Doudna to take her IP to the competing Intellia, and Swiss rival CRISPR Therapeutics has conflicting claims of its own. -- Damian Garde

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