Cell/Gene Tx channel feed

Spun out of George Church's lab, this biotech up­start is map­ping the AAV uni­verse for No­var­tis, Sarep­ta to gaze

In a few days, through a series of video conferences, gene therapy researchers around the world will be presenting their latest findings at the virtual annual meeting of the American Society of Gene & Cell Therapy. Almost every discussion will feature a topic that has been central to the existence of the field but continues to perplex experts as they seek to refine the modality: the delivery of a gene to the tissue where it’s needed to fix disease.

FDA is­n't lift­ing the clin­i­cal hold on Sol­id Bio's Duchenne MD gene ther­a­py just yet — but is there a sil­ver lin­ing?

It may now be close to a year before Solid Bio can resume human testing on its lead gene therapy program.

The clinical hold that the FDA slapped on the program last November in the wake of a severe adverse event marked the second such interruption for the Phase I/II IGNITE DMD study, which was testing SGT-001 in a small cohort of patients with Duchenne muscular dystrophy. While Solid Bio resolved the first hold within three months, this time around regulators demanded more data.

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'Deeply com­mit­ted' Jim Wil­son pours more of his life's work in­to Pas­sage Bio — lock­ing in more gene ther­a­py pro­grams, new tech

From Passage Bio’s inception last February to its IPO filing a year later, its close relationship with co-founder Jim Wilson’s Gene Therapy Program at the University of Pennsylvania has always been touted as a major strength. Now the biotech is tying an even tighter knot with its academic partner.

In a deal expansion, Passage nabbed the option to license five more programs coming out of Wilson’s lab and extended the exercise period by three years to 2025. If all the candidates end up making the cut, they will feed 17 new treatments for rare monogenic diseases all the way through clinical development.

JJ Bienaimé, BioMarin CEO (via YouTube)

Bio­Marin broad­ens its gene ther­a­py hori­zons with a new R&D al­liance in rare car­dio cas­es

BioMarin has just struck a deal to back a Swiss biotech startup with some deep ties to top research institution UCL in London as it beefs up the swelling gene therapy portion of the pipeline.

We don’t have any terms to deal with, just the knowledge that BioMarin CEO JJ Bienaimé saw enough of DiNAQOR’s work to invest in the company as it licenses their lead preclinical program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy, while collaborating on the rest of the pipeline.

No­var­tis spin­out resTOR­bio re­verse merges with T cell biotech af­ter big PhI­II fail­ure

The end of their lead program will, it turns out, spell the end for Novartis spinout resTORbio.

Shares for the once well-funded anti-aging biotech crumbled after its lead program failed a Phase III trial last year. The company said they would shift focus to an early stage Parkinson’s candidate, but that looked like a long shot at the time, and today, the company announced they will reverse merge with Adicet Bio.

RA Session (Taysha)

In the heart of Texas, the AveX­is crew gath­ers for an­oth­er go at gene ther­a­py with $30M and a pow­er­house aca­d­e­m­ic pact to start

The same week RA Session II closed the seed financing for his latest gene therapy startup — back in early March — the Dow dropped a couple of thousand points.

“If you had to ask me, was I worried? Absolutely. I think I wouldn’t be human if I wasn’t,” he said.

But Session had a lot of certainty to balance out all the uncertainty of launching a new biotech during a pandemic. There was the management team comprising some longtime colleagues at Reata and AveXis; there was PBM Capital, AveXis’ first institutional investor, as well as former CEO Sean Nolan, who together provided $30 million to get things started; then there was the foundational partnership with Steven Gray and Berge Minassian at UT Southwestern’s gene therapy program, complete with licenses to 15 AAV9-based assets.

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Hav­ing sold its cor­po­rate self, Toca­gen auc­tions off sci­en­tif­ic plat­form to San Diego biotech

With the dermatitis specialists at Forte Biosciences now inhabiting Tocagen’s corporate skeleton, a small San Diego biotech has won rights to its scientific platform.

Denovo Biopharma, a biotech based in San Diego and backed largely by investors in China, will acquire Tocagen’s retro-viral replicating platform, including their former lead gene therapy for brain cancer. The drug, now rebranded as DB107, failed a Phase III clinical trial last year, but Denovo said they saw promising signs in a subset of patients and will use their biomarker platform to select patients they believe the therapy will work on.

Analy­sis shows Swiss biotech in­dex out­shines Nas­daq coun­ter­part, but coro­n­avirus chal­lenges are uni­ver­sal

In 2004, the Swiss Stock Exchange spawned its biotech index — the Swiss Bio+Medtech Index (SXI) — as the life sciences sector, headlined by large successful biopharma companies Roche and Novartis, took center stage. In the last five years through January 2020, SXI has produced returns of 59%, outperforming both the Swiss Performance Index (31%) and the NASDAQ Biotechnology Index (35%), a new analysis shows.

Mer­ck KGaA adds to gene ther­a­py man­u­fac­tur­ing boom with €100M fa­cil­i­ty in Cal­i­for­nia

Add a new, €100 million California facility to the growing gene therapy infrastructure.

Merck KGaA announced they are opening a second gene therapy and viral vector factory in Carlsbad, California. The new center will be just one point in the global supply chain Big Pharma is rapidly erecting to keep manufacturing capacity for the new technology at pace with clinical development. Over the past year, Novartis, PTC Therapeutics, Pfizer and Vertex each announced or opened new facilities in Switzerland, North Carolina and New Jersey that will help build gene therapy.

Dy­nacure se­cures $55M as their Io­n­is-de­vel­oped drug moves in­to clin­ic

A month after dosing their first ever patient with their Ionis-licensed tech, Dynacure has announced a new round of funding to advance the trial.

The French biotech announced €50 million ($55 million) in Series C funding, led by Perceptive Advisors. Although listed as a Series C, it is the second tranche of cash the company has ever announced, after their initial $55 million raise in 2018. The cash will float Dynacure through its Phase I/II trial in a class of neuromuscular diseases known as myotubular and centronuclear myopathies.