Seamless Therapeutics CEO Anne-Kristin Heninger and CSO Felix Lansing

Ger­man biotech launch­es with ‘seam­less’ gene edit­ing plat­form built on evolv­ing en­zymes

Seam­less Ther­a­peu­tics has launched with €11.8 mil­lion (rough­ly $12.5 mil­lion) in seed fund­ing to build out a gene edit­ing plat­form.

As more and more gene edit­ing tech­niques are be­ing de­vel­oped — start­ing with CRISPR/Cas9 to base and prime edit­ing, and more re­cent­ly, PASTE — Seam­less is try­ing to evolve, lit­er­al­ly, an ap­proach that dates back decades.

The Dres­den, Ger­many-based start­up is de­vel­op­ing so-called “de­sign­er re­com­bi­nas­es” that can po­ten­tial­ly ed­it large chunks of genes. Re­com­bi­nas­es are en­zymes that can ex­cise, in­vert, or even in­sert a DNA se­quence, but typ­i­cal­ly they on­ly work via very spe­cif­ic tar­get se­quences. How­ev­er, by con­tin­u­ous­ly evolv­ing these re­com­bi­nas­es, Seam­less is try­ing to de­vel­op re­com­bi­nas­es that work at the se­quences it wants — those that are found in hu­man genes.

The biotech stems from Frank Buch­holz’s lab at the Uni­ver­si­ty of Dres­den, where CSO Fe­lix Lans­ing was a grad­u­ate stu­dent and lat­er a post­doc. It orig­i­nal­ly start­ed out near­ly four years ago as RecTech, bankrolled by a €3.4 mil­lion grant from a Ger­man gov­ern­ment ini­tia­tive for life sci­ence star­tups, but of­fi­cial­ly launched as Seam­less at the start of this year. At the helm is CEO Anne-Kristin Heninger, who was a sci­en­tif­ic project man­ag­er for RecTech at its in­cep­tion and has been in charge of the op­er­a­tions and the busi­ness side of the start­up.

Welling­ton Part­ners and For­bion are the ven­ture cap­i­tal back­ers be­hind the biotech. Part of Seam­less’ seed fund­ing al­so in­cludes a new €3.8 mil­lion grant from the Ger­man gov­ern­ment.

For many years, re­com­bi­nas­es have been a sta­ple of ba­sic sci­ence re­search used to study how genes are ex­pressed, but now Seam­less and oth­ers are lever­ag­ing them for fu­ture med­i­cines. Prime Med­i­cine, found­ed by the Broad In­sti­tute’s David Liu, is work­ing on a tech­nique called PAS­SIGE that us­es a prime ed­i­tor to put a tar­get site where it wants in the DNA, so a re­com­bi­nase can then swoop in and ed­it the gene. Stealth biotech Tome Bio­sciences, co-found­ed by MIT fel­lows Omar Abu­dayyeh and Jonathan Gooten­berg, house PASTE, a tech­nol­o­gy that fus­es the re­com­bi­nase and the parts of a prime ed­i­tor to­geth­er. Seam­less, on the oth­er hand, is point­ing the re­com­bi­nas­es to their tar­gets by al­ter­ing the re­com­bi­nas­es them­selves.

Seam­less’ de­sign­er re­com­bi­nas­es take time to make. “When I start­ed in 2016 with my PhD, it took me one and a half years to cre­ate my first pair of de­sign­er re­com­bi­nas­es,” Lans­ing said. “But then, ramp­ing up the tech­nol­o­gy and un­der­stand­ing it, we are now down to a cou­ple of months for mak­ing a new en­zyme.”

“We re­al­ly had em­pha­sis put on speed­ing up the process of gen­er­at­ing [new re­com­bi­nas­es] be­cause that will tru­ly un­lock the us­age in a broad­er as­pect and be­cause we would like to test many things that are very ver­sa­tile,” he added.

Seam­less is still in its ear­ly stages, and Heninger said that next steps will be to ma­ture the plat­form and build out Seam­less’ pipeline. She said it was too ear­ly to put a time­line on mile­stones for the com­pa­ny, like when it would be in the clin­ic.

First, the biotech is like­ly to work on ex vi­vo ap­proach­es as it looks in­to po­ten­tial de­liv­ery tech­nolo­gies for in vi­vo ther­a­pies, Lans­ing said. On de­liv­ery, Heninger added that the biotech may look for po­ten­tial col­lab­o­ra­tors.

De­liv­er­ing gene ed­i­tors pos­es a ma­jor lim­it­ing fac­tor for what ther­a­pies can be made suc­cess­ful­ly, giv­ing rise to a new slate of biotech com­pa­nies fo­cus­ing specif­i­cal­ly on just that, in­clud­ing Feng Zhang’s Aera Ther­a­peu­tics, which launched last month. Liu and Kei­th Joung are the sci­en­tif­ic founders be­hind an­oth­er de­liv­ery biotech called Nvel­op Ther­a­peu­tics.

When asked how Seam­less’ plat­form com­pared to oth­er gene edit­ing tools, Heninger said, “It’s im­por­tant to point out to maybe not com­pare the tech­nolo­gies. Rather say, what can we ad­dress best with our tech­nol­o­gy? And they have good in­di­ca­tions they can ad­dress with their tech­nolo­gies. I would rather think about it that way.”

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

Man­u­fac­tur­ing roundup: Catal­ent to pro­duce low-cost ver­sion of nalox­one; CSL opens R&D site

Catalent will be manufacturing a low-cost version of the opioid overdose treatment naloxone as part of a contract with Harm Reduction Therapeutics.

Catalent plans to manufacture the treatment at its facility in Morrisville, NC. No financial details on the deal were disclosed.

Harm Reduction was granted priority review status for the NDA on its spray last year. The company has been working on a naloxone product since 2017. It is anticipating approval in July of this year and a US launch in early 2024.

As­pen looks to re­bound in pro­duc­tion and rev­enue af­ter Covid-19

Last year, South African-based vaccine manufacturer Aspen Pharmacare was facing reports that it had not received a single order for its manufactured Covid-19 shots and that manufacturing lines were sitting idle. But now the vaccine producer is looking to turn things around.

Aspen’s disclosure of its financial results in March unveiled that manufacturing revenue had decreased by 12% to R 603 million ($33.8 million), which Lorraine Hill, Aspen Group’s COO, said is attributable to lower Covid vaccine sales.

Ribbon cutting ceremony for Thermo Fisher's new cell therapy manufacturing site in San Francisco

Ther­mo Fish­er moves on cam­pus with new cell man­u­fac­tur­ing site in San Fran­cis­co

Thermo Fisher Scientific is putting down more roots in the Bay Area.

The manufacturer opened the doors to a new cell therapy manufacturing facility next to the University of California-San Francisco Medical Center’s Mission Bay campus and on the university’s campus.

UCSF and Thermo Fisher have had a partnership since 2021, with the new site focusing on manufacturing cell therapeutics for certain cancers, including glioblastoma and multiple myeloma. The new site plans to use Thermo Fisher’s expertise in manufacturing services to help UCSF accelerate the development of cell therapies and eventually get them into the clinic, said Dan Herring, the general manager of cell therapy services at Thermo Fisher, in an interview with Endpoints News.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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FDA grants full ap­proval to Keytru­da in tu­mor-ag­nos­tic set­ting; Can­del paus­es tri­al en­roll­ment

In a first, Merck has secured a full approval for Keytruda in a tumor agnostic setting — as a treatment for any unresectable or metastatic solid tumors that are classified as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR).

The FDA granted Keytruda accelerated approval in this indication in 2017, and GSK’s Jemperli followed suit in 2021. But now it’s converted to a full approval for Keytruda. Before prescribing, doctors would have to make sure patients carry this biomarker, using an FDA-approved test.